Produits et services

Pharmaceuticals; pharmaceutical preparations and substances; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein or nucleic acids or viral vectors for therapy; medicine; medical preparations and articles; biological preparations for medical purposes; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; biological preparations comprising exosome; biological preparations comprising a therapeutic target protein-loaded, nucleic acid-loaded or virus-loaded exosome; biological preparations comprising exosome for medical use; biological reagents for medical use; mixed biological preparations for medical purposes; mixed biological preparations for the prevention and treatment of infectious diseases; extracellular vesicle-based nucleic acid preparations for therapeutical use for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; gene therapy products, namely, gene delivery pharmaceuticals; pharmaceutical preparations for gene therapy, gene editing, and genome editing; pharmaceutical preparations for use in gene therapies for the treatment of genetic diseases and disorders. Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; research and analysis services; biological research; clinical research; providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood; scientific investigations for medical purposes; scientific laboratory services; medical laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; laboratory diagnostics; genetic engineering services; genetic research; research in the field of gene editing technology; research in the field of DNA mutation; information, advisory and consultancy services relating to the aforesaid. Medical services; medical and pharmaceutical consultancy; medical diagnostics; medical analysis and examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; information, advisory and consultancy services relating to the aforesaid.

Abrégé

Polynucleotides and polynucleotide sequences encoding phenylalanine hydroxylase (PAH), expression cassettes, vectors, viral particles, Extracellular Vesicles (EVs) and pharmaceutical compositions comprising the same, and uses thereof, for example to treat diseases and disorders associated with low levels or reduced activity of PAH, such as Phenylketonuria (PKU).

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C07K 14/00 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés
• C12N 15/86 - Vecteurs viraux

Abrégé

Modified guide RNAs for EV-mediated delivery of gene editing machinery, complexes, EVs, EV producer cells and pharmaceutical compositions comprising the same, and uses thereof, for example to treat genetic diseases and disorders.

Classes IPC  ?

• C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
• C07K 19/00 - Peptides hybrides

Abrégé

A modified Extracellular Vesicle (EV), preferably an exosome, comprising an albumin protein or fragment (e.g., an albumin domain) present on the surface of the EV membrane, preferably as part of a fusion protein (e.g., a transmembrane protein). The EV exhibits an improved circulating half-life, stability and shelf-life (both formulation and storage). Methods of making said EVs, as well as pharmaceutical compositions and their use.

Classes IPC  ?

• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• C12N 5/0775 - Cellules souches mésenchymateusesCellules souches dérivées du tissu adipeux
• C12P 21/02 - Préparation de peptides ou de protéines comportant une séquence connue de plusieurs amino-acides, p. ex. glutathion

Produits et services

Pharmaceuticals; pharmaceutical preparations and substances; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein or nucleic acids or viral vectors for therapy; medicine; medical preparations and articles; biological preparations for medical purposes; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; biological preparations comprising exosome; biological preparations comprising a therapeutic target protein-loaded, nucleic acid-loaded or virus-loaded exosome; biological preparations comprising exosome for medical use; biological reagents for medical use; mixed biological preparations for medical purposes; mixed biological preparations for the prevention and treatment of infectious diseases; extracellular vesicle-based nucleic acid preparations for therapeutical use for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; gene therapy products, namely, gene delivery pharmaceuticals; pharmaceutical preparations for gene therapy, gene editing, and genome editing; pharmaceutical preparations for use in gene therapies for the treatment of genetic diseases and disorders. Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; research and analysis services; biological research; clinical research; providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood; scientific investigations for medical purposes; scientific laboratory services; medical laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; laboratory diagnostics; genetic engineering services; genetic research; research in the field of gene editing technology; research in the field of DNA mutation; information, advisory and consultancy services relating to the aforesaid. Medical services; medical and pharmaceutical consultancy; medical diagnostics; medical analysis and examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; information, advisory and consultancy services relating to the aforesaid.

Produits et services

Pharmaceuticals; pharmaceutical preparations and substances; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein or nucleic acids or viral vectors for therapy; medicine; medical preparations and articles; biological preparations for medical purposes; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; biological preparations comprising exosome; biological preparations comprising a therapeutic target protein-loaded, nucleic acid-loaded or virus-loaded exosome; biological preparations comprising exosome for use in the manufacture of pharmaceuticals; biological reagents for medical use; mixed biological preparations for medical purposes; mixed biological preparations for the prevention and treatment of infectious diseases; diagnostic reagents for medical use; medical diagnostic reagents comprising exosome; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, EV-based nucleic acid therapeutics for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders. Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; research and analysis services; biological research; clinical research; providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood; scientific investigations for medical purposes; scientific laboratory services; medical laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; laboratory diagnostics research services; information, advisory and consultancy services relating to the aforesaid. Medical services; medical and pharmaceutical consultancy; medical diagnostics; medical analysis and examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; information, advisory and consultancy services relating to the aforesaid.

Abrégé

The present invention pertains to a genetically engineered extracellular vesicle comprising an improved scaffold protein and a protein of interest. Polypeptide constructs, polynucleotide constructs, cells, pharmaceutical compositions and methods relating to the same are also disclosed.

Classes IPC  ?

• C12N 9/22 - Ribonucléases
• A61K 9/50 - Microcapsules
• A61K 38/00 - Préparations médicinales contenant des peptides
• C07K 14/435 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains

Abrégé

The present invention relates to engineered extracellular vesicles (EVs) as a therapeutic modality for the treatment of various severe diseases. More specifically, the invention relates to a novel approach to manufacturing engineered EVs which extends their half-life and alters their biodistribution, resulting in a novel therapeutic modality able to carry various types of drugs suitable for application in not only genetic diseases but more broadly across essentially all therapeutic areas.

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 9/50 - Microcapsules
• C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
• C07K 14/54 - Interleukines [IL]
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• C07K 14/715 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des cytokinesRécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des lymphokinesRécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des interférons
• C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques

Abrégé

The present invention pertains to extracellular vesicles (EVs), wherein the EVs comprise domains present on the surface of the EV, which modify the circulation time of such EVs. Such EVs display improved pharmacokinetics and are therefore useful as therapeutics. Such EVs may also be useful for affinity purification of the EV drug substance.

Classes IPC  ?

• A61K 9/50 - Microcapsules
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• C07K 14/765 - Sérum albumine, p. ex. HSA
• A61K 35/12 - Substances provenant de mammifèresCompositions comprenant des tissus ou des cellules non spécifiésCompositions comprenant des cellules souches non embryonnairesCellules génétiquement modifiées

Abrégé

The present disclosure relates compartmental administration of modified extracellular vesicles, e.g., exosomes. In some aspects, the extracellular vesicle, e.g., exosome, comprises a biologically active molecule and a targeting moiety.

Classes IPC  ?

• A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique
• A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier

Produits et services

Pharmaceuticals for the treatment of cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; pharmaceutical preparations and substances for the treatment of cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein or nucleic acids or viral vectors for therapy; medicine for the treatment of cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; medical preparations for the treatment of cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations for medical purposes for the treatment of cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations for the treatment of cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies in the nature of mitochondrial disease, joint and ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; biological preparations comprising exosome for the treatment of cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations comprising a therapeutic target protein-loaded, nucleic acid-loaded or virus-loaded exosome for the treatment of genetic diseases, respiratory diseases, or cancer; biological preparations comprising exosome sold as a component of pharmaceuticals for the treatment of cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological reagents for medical use; mixed biological preparations for medical purposes for the prevention and treatment of cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; mixed biological preparations for the prevention and treatment of infectious diseases; diagnostic reagents used in the fields of biological, genetic engineering and pharmaceutical research; diagnostic reagents comprising exosome used in the fields of biological, genetic engineering and pharmaceutical research; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, extracellular vesicle (EV)-based nucleic acid therapeutics for the prevention and treatment of infections or cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies in the nature of mitochondrial disease, joint and ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; all of the foregoing excluding such goods or services in the fields of constipation, gut health, weight loss, appetite suppressants, dietetic preparations, and nutrition Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; research and analysis services in the field of pharmaceuticals; biological research; clinical research in the field of clinical research in the field of transformational therapeutics; providing medical research and scientific research information in the field of filtration of exosomes from cultured cells or blood; scientific investigations for medical purposes; scientific laboratory services; medical research laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; medical laboratory diagnostics research services; information, advisory and consultancy services relating to the aforesaid; none of the foregoing in the fields of information technology and information technology systems; all of the foregoing excluding such goods or services in the fields of constipation, gut health, weight loss, appetite suppressants, dietetic preparations, and nutrition Pharmaceutical consultancy; medical diagnostic services; medical analysis for the diagnosis and treatment of persons and medical examinations; medical treatment services; medical screening; medical monitoring, testing, and inspection services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases; medical monitoring, testing, and inspection services relating to the diagnosis and treatment of cancer, diabetes, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint and ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; information, advisory and consultancy services relating to the aforesaid

Produits et services

Pharmaceuticals; pharmaceutical preparations and substances; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein or nucleic acids or viral vectors for therapy; medicine; medical preparations and articles; biological preparations for medical purposes; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; biological preparations comprising exosome; biological preparations comprising a therapeutic target protein-loaded, nucleic acid-loaded or virus-loaded exosome; biological preparations comprising exosome for use in the manufacture of pharmaceuticals; biological reagents for medical use; mixed biological preparations for medical purposes; mixed biological preparations for the prevention and treatment of infectious diseases; diagnostic reagents used in the fields of biological, genetic engineering and pharmaceutical research; diagnostic reagents comprising exosome used in the fields of biological, genetic engineering and pharmaceutical research; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, EV-based nucleic acid therapeutics for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; Diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders. Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; biological research; clinical research; providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood; scientific investigations for medical purposes; scientific laboratory services; medical laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome and dementia; research and development in the field of biotherapeutics for the treatment of other aging associated pathologies such as mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; Laboratory services for analytical testing; information, advisory and consultancy services relating to the aforesaid. Medical services; medical and pharmaceutical consultancy; medical diagnostics; medical analysis and examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome and dementia; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; information, advisory and consultancy services relating to the aforesaid.

Abrégé

The present invention pertains to a new nanoparticle-like delivery system for intracellular delivery of cargo molecules such as nucleic acids, ribonucleoproteins and extracellular vesicles.

Classes IPC  ?

• C07K 7/06 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 5 à 11 amino-acides
• C07K 7/08 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 12 à 20 amino-acides
• C07K 14/00 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit

Produits et services

Pharmaceuticals for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; pharmaceutical preparations and substances for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein or nucleic acids or viral vectors for therapy; medicine for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; medical preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations for medical purposes for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies in the nature of mitochondrial disease, joint and ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; biological preparations comprising exosome for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations comprising a therapeutic target protein-loaded, nucleic acid-loaded or virus-loaded exosome for the treatment of genetic diseases, respiratory diseases, or cancer; biological preparations comprising exosome sold as a component of pharmaceuticals for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological reagents for medical use; mixed biological preparations for medical purposes for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; mixed biological preparations for the prevention and treatment of infectious diseases; diagnostic reagents for medical use; medical diagnostic reagents comprising exosome; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, extracellular vesicle (EV)-based nucleic acid therapeutics for the prevention and treatment of infections or disease, namely, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies in the nature of mitochondrial disease, joint and ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; research and analysis services, namely, those in the field of pharmaceuticals; biological research; clinical research in the field of transformational therapeutics; providing medical research and scientific research information in the field of filtration of exosomes from cultured cells or blood; scientific investigations for medical purposes; scientific laboratory services; medical research laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; medical laboratory diagnostics research services; information, advisory and consultancy services relating to the aforesaid Medical services; medical and pharmaceutical consultancy; medical diagnostic services; medical analysis for the diagnosis and treatment of persons and medical examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint and ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; information, advisory and consultancy services relating to the aforesaid

Abrégé

The present invention pertains to an inventive release mechanism for extracellular vesicle (EV)-mediated intracellular and intramembrane delivery of therapeutic polypeptides. More specifically, the invention relates to EVs comprising polypeptide constructs which comprise a therapeutic polypeptide releasably attached to an exosomal polypeptide. Furthermore, the present invention pertains to manufacturing methods, pharmaceutical compositions, medical uses and applications, and various other embodiments related to the inventive EVs.

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61K 47/65 - Séquences de liaison, liants ou bras-espaceurs peptidiques, p. ex. séquences de liaison peptidiques vulnérable aux protéases
• A61K 41/00 - Préparations médicinales obtenues par traitement de substances par énergie ondulatoire ou par rayonnement corpusculaire
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• A61K 47/00 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif
• A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique

Abrégé

An engineered extracellular vesicle (EV) for delivering a bioactive cargo, the engineered EV comprising an endosomal escape enhancer and a cargo. Plasmids, polynucleotide sequences, cells, methods of making and analysis, pharmaceutical compositions and uses thereof.

Classes IPC  ?

• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit

Abrégé

The present invention pertains to inter alia methods for purifying extracellular vesicles including exposing a sample comprising at least one extracellular vesicle to ultrafiltration; and exposing the sample following the ultrafiltration in step (i) to size exclusion liquid chromatography.

Classes IPC  ?

• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• F01C 21/10 - Organes externes coopérant avec des pistons rotatifsCarcasses d'enveloppes
• F01C 1/344 - "Machines" ou machines motrices à piston rotatif ayant des caractéristiques couvertes par au moins deux des groupes , , , ou par l'un de ces groupes associées à un autre type de mouvement entre les organes coopérants ayant à la fois le mouvement défini dans le groupe ou et un mouvement alternatif entre les organes coopérants comportant des organes obturateurs se déplaçant par rapport à l'organe interne
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 47/62 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant une protéine, un peptide ou un acide polyaminé
• A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
• A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains

Produits et services

Pharmaceuticals, namely, drug delivery agents in the form of exosomes consisting of compounds that facilitate the delivery of a wide range of pharmaceutical preparations; pharmaceutical preparations, namely, drug delivery agents in the form of exosomes that facilitate the delivery of pharmaceutical preparations; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use, namely, for the release of a wide variety of therapeutic agents; pharmaceutical preparations, namely, engineered exosomes to deliver pharmaceutical preparations in the nature of protein for genetic therapy; medicine, namely, drug delivery agents in the form of exosomes that facilitate the delivery of pharmaceutical preparations; medical preparations, namely, drug delivery agents in the form of exosomes that facilitate the delivery of pharmaceutical preparations; biological preparations for medical purposes, namely, for the treatment of cancer, diabetes, metabolic syndrome, dementia, and aging associated pathologies in the nature of mitochondrial disease, joint and ligament strains and tears, muscle atrophy, and muscular dystrophy; biological preparations for the treatment of cancer, diabetes, metabolic syndrome, dementia, and aging associated pathologies in the nature of mitochondrial disease, joint and ligament strains and tears, muscle atrophy, muscular dystrophy; biological preparations comprising exosomes, namely, drug delivery agents in the form of exosomes that facilitate the delivery of pharmaceutical preparations, for the treatment of cancer, diabetes, metabolic syndrome, dementia, and aging associated pathologies in the nature of mitochondrial disease, joint and ligament strains and tears, muscle atrophy, and muscular dystrophy; biological preparations comprising a therapeutic target protein-loaded exosome that facilitates the delivery of pharmaceutical preparations, for the treatment of cancer, diabetes, metabolic syndrome, dementia, and aging associated pathologies in the nature of mitochondrial disease, joint and ligament strains and tears, muscle atrophy, and muscular dystrophy; biological preparations comprising exosomes for pharmaceutical purposes that facilitate the delivery of pharmaceutical preparations, for the treatment of cancer, diabetes, metabolic syndrome, dementia, and aging associated pathologies in the nature of mitochondrial disease, joint and ligament strains and tears, muscle atrophy, and muscular dystrophy; biological reagents for medical use, namely, drug delivery agents in the form of exosomes that facilitate the delivery of pharmaceutical preparations; mixed biological preparations for medical purposes, namely, for the prevention and treatment of infectious diseases; mixed biological preparations for the prevention and treatment of infectious diseases; diagnostic reagents used in the fields of biological, genetic engineering and pharmaceutical research; diagnostic reagents comprising exosome used in the fields of biological, genetic engineering and pharmaceutical research; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, extracellular vesicle (EV)-based nucleic acid therapeutics for the prevention and treatment of infections or infectious diseases; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, metabolic syndrome, dementia, and aging associated pathologies, namely, mitochondrial disease, joint and ligament strains and tears, muscle atrophy, and muscular dystrophy; all of the foregoing excluding such goods in the fields of digestive diseases, gastrointestinal diseases, constipation, gut health, weight loss, appetite suppressants, dietetic preparations, nutrition, and enzymes Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; scientific research and analysis services in the field of pharmaceuticals; biological research; clinical research in the field of transformational therapeutics; providing medical research and scientific research information in the field of filtration of exosomes from blood; scientific investigations for medical purposes; scientific laboratory services; medical research laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of infectious diseases and metabolic disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; all of the foregoing excluding such services in the fields of digestive diseases, gastrointestinal diseases, constipation, gut health, weight loss, appetite suppressants, dietetic preparations, nutrition, and enzymes

Abrégé

The present invention relates to engineered extracellular vesicles (EVs) as a therapeutic modality for the treatment of various severe diseases. More specifically, the invention relates to delivery of nucleic acid cargos by a peptide delivery vector which enables a novel approach to manufacturing engineered EVs which extends their half-life and alters their biodistribution, resulting in a novel therapeutic modality able to carry various types of drugs suitable for application in not only genetic diseases but more broadly across essentially all therapeutic areas.

Classes IPC  ?

• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques

Abrégé

A modified Extracellular Vesicle (EV), preferably an exosome, comprising an albumin protein or fragment (e.g., an albumin domain) present on the surface of the EV membrane, preferably as part of a fusion protein (e.g., a transmembrane protein). The EV exhibits an improved circulating half-life, stability and shelf-life (both formulation and storage). Methods of making said EVs, as well as pharmaceutical compositions and their use.

Classes IPC  ?

• A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
• A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit

Abrégé

The present disclosure relates to extracellular vesicles, e.g., exosomes, comprising an AAV and a scaffold protein. In some aspects, the AAV is in the lumen of the extracellular vesicle. In some aspects, the AAV is associated with the luminal surface of the extracellular vesicle. In some aspects, the AAV is associated with the exterior surface of the extracellular vesicle. Also provided herein are methods for producing the exosomes and methods for using the exosomes to treat and/or prevent diseases or disorders.

Classes IPC  ?

• A61K 35/761 - Adénovirus
• A61K 47/42 - ProtéinesPolypeptidesLeurs produits de dégradationLeurs dérivés p. ex. albumine, gélatine ou zéine
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
• A61P 27/02 - Agents ophtalmiques
• A61P 3/10 - Médicaments pour le traitement des troubles du métabolisme de l'homéostase du glucose de l'hyperglycémie, p. ex. antidiabétiques
• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C12N 15/86 - Vecteurs viraux

Abrégé

The present invention pertains to extracellular vesicle (EV) therapeutics, wherein the EVs comprise nucleic acid (NA)-based therapeutics such as mRNAs, circular RNAs, miRNAs, shRNAs, and/or DNA molecules. The NA therapeutics are loaded into EVs using inventive protein and NA engineering strategies which stabilize the cargo NAs and enhance loading into EVs, thereby enhancing therapeutic activity of the cargo NA molecules half-life.

Classes IPC  ?

• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 9/50 - Microcapsules
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
• C12N 9/22 - Ribonucléases
• C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
• C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques

Abrégé

The present invention relates to methods for loading extracellular vesicles (EVs) with a pharmacological agent. The invention discloses the use of cell-penetrating peptides as carriers into EVs, using either a non-covalent or covalent loading approach. Furthermore, the present invention pertains to medical uses and compositions comprising such pharmacological agent-loaded EVs.

Classes IPC  ?

• A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 47/61 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique macromoléculaire, p. ex. une molécule oligomérique, polymérique ou dendrimérique le composé organique macromoléculaire étant un polysaccharide ou l’un de ses dérivés
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
• A61K 9/50 - Microcapsules
• C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques

Abrégé

The present invention relates to engineered extracellular vesicles (EVs) as a therapeutic modality for the treatment of various severe diseases. More specifically, the invention relates to a novel approach to manufacturing engineered EVs which extends their half-life and alters their biodistribution, resulting in a novel therapeutic modality able to carry various types of drugs suitable for application in not only genetic diseases but more broadly across essentially all therapeutic areas.

Classes IPC  ?

• C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques
• A61K 47/66 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant une protéine, un peptide ou un acide polyaminé l’agent de modification étant un système de pré-ciblage impliquant un peptide ou une protéine pour cibler des cellules spécifiques
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit

Abrégé

The present invention relates to engineered extracellular vesicles (EVs) as a therapeutic modality for the treatment of various severe diseases. More specifically, the invention relates to a novel approach to manufacturing engineered EVs which extends their half-life and alters their biodistribution, resulting in a novel therapeutic modality able to carry various types of drugs suitable for application in not only genetic diseases but more broadly across essentially all therapeutic areas.

Classes IPC  ?

• C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques
• A61K 47/66 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant une protéine, un peptide ou un acide polyaminé l’agent de modification étant un système de pré-ciblage impliquant un peptide ou une protéine pour cibler des cellules spécifiques
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit

Abrégé

The present invention pertains to extracellular vesicle (EV) therapeutics, wherein the EVs are coated with proteins containing Fc domains (such as antibodies) for i.a. targeting and therapeutic applications. The coating of EVs is achieved through inventive protein engineering of EV polypeptides. The present invention thus relates to methods for coating of EVs, EVs per se, as well as pharmaceutical compositions and medical applications of such EVs coated with Fc containing proteins.

Classes IPC  ?

• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique
• A61K 47/24 - Composés organiques, p. ex. hydrocarbures naturels ou synthétiques, polyoléfines, huile minérale, gelée de pétrole ou ozocérite contenant des atomes autres que des atomes de carbone, d'hydrogène, d'oxygène, d'halogènes, d'azote ou de soufre, p. ex. cyclométhicone ou phospholipides
• C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères

Produits et services

Pharmaceuticals; pharmaceutical preparations and substances; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein or nucleic acids or viral vectors for therapy; medicine; medical preparations and articles; biological preparations for medical purposes; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; biological preparations comprising exosome; biological preparations comprising a therapeutic target protein-loaded, nucleic acid-loaded or virus-loaded exosome; biological reagents for medical use; mixed biological preparations for medical purposes; mixed biological preparations for the prevention and treatment of infectious diseases; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, EV-based nucleic acid therapeutics for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders. Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; scientific research and analysis services; biological research; clinical research; providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood; scientific investigations for medical purposes; scientific laboratory services; medical laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; laboratory diagnostics; information, advisory and consultancy services relating to the aforesaid. Medical services; medical and pharmaceutical consultancy; medical diagnostics; medical analysis and examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; information, advisory and consultancy services relating to the aforesaid.

Produits et services

Pharmaceuticals; pharmaceutical preparations and substances; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein or nucleic acids or viral vectors for therapy; medicine; medical preparations and articles; biological preparations for medical purposes; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; biological preparations comprising exosome; biological preparations comprising a therapeutic target protein-loaded, nucleic acid-loaded or virus-loaded exosome; biological reagents for medical use; mixed biological preparations for medical purposes; mixed biological preparations for the prevention and treatment of infectious diseases; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, EV-based nucleic acid therapeutics for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders. Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; scientific research and analysis services; biological research; clinical research; providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood; scientific investigations for medical purposes; scientific laboratory services; medical laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; laboratory diagnostics; information, advisory and consultancy services relating to the aforesaid. Medical services; medical and pharmaceutical consultancy; medical diagnostics; medical analysis and examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; information, advisory and consultancy services relating to the aforesaid.

Produits et services

Pharmaceuticals; pharmaceutical preparations and substances; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein or nucleic acids or viral vectors for therapy; medicine; medical preparations and articles; biological preparations for medical purposes; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; biological preparations comprising exosome; biological preparations comprising a therapeutic target protein-loaded, nucleic acid-loaded or virus-loaded exosome; biological reagents for medical use; mixed biological preparations for medical purposes; mixed biological preparations for the prevention and treatment of infectious diseases; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, EV-based nucleic acid therapeutics for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders. Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; scientific research and analysis services; biological research; clinical research; providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood; scientific investigations for medical purposes; scientific laboratory services; medical laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; laboratory diagnostics; information, advisory and consultancy services relating to the aforesaid. Medical services; medical and pharmaceutical consultancy; medical diagnostics; medical analysis and examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; information, advisory and consultancy services relating to the aforesaid.

Abrégé

The present invention pertains to extracellular vesicles (EVs), wherein the EVs comprise domains present on the surface of the EV, which modify the circulation time of such EVs. Such EVs display improved pharmacokinetics and are therefore useful as therapeutics. Such EVs may also be useful for affinity purification of the EV drug substance.

Classes IPC  ?

• A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique

Abrégé

The present invention pertains to extracellular vesicles (EVs), wherein the EVs comprise domains present on the surface of the EV, which modify the circulation time of such EVs. Such EVs display improved pharmacokinetics and are therefore useful as therapeutics. Such EVs may also be useful for affinity purification of the EV drug substance.

Classes IPC  ?

• A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
• A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit

Abrégé

The present invention relates to engineered extracellular vesicles (EVs) as a novel therapeutic approach to treating urea cycle disorders. More specifically, the invention relates to the use of various protein engineering and nucleic acid engineering strategies for improving loading of urea cycle proteins or nucleic acids encoding urea cycle proteins into EVs and targeting of the resultant EVs to tissues and organs of interest.

Classes IPC  ?

• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire

Abrégé

The present invention pertains to extracellular vesicle (EV) therapeutics, wherein the EVs comprise nucleic acid (NA)-based therapeutics such as mRNAs, circular RNAs, miRNAs, shRNAs, circular RNA and/or DNA molecules. The NA therapeutics are loaded into EVs using inventive engineering protein and NA engineering strategies to enhance loading into EVs and to facilitate release of the NA cargo molecules inside target cells.

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
• C12N 9/22 - Ribonucléases
• C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques
• A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique

Abrégé

The present disclosure relates compartmental administration of modified extracellular vesicles, e.g., exosomes. In some aspects, the extracellular vesicle, e.g., exosome, comprises a biologically active molecule and a targeting moiety.

Classes IPC  ?

• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• A61K 47/62 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant une protéine, un peptide ou un acide polyaminé
• A61K 47/68 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un anticorps, une immunoglobuline ou son fragment, p. ex. un fragment Fc
• A61K 9/133 - Vésicules unilamellaires
• A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides
• A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
• A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
• C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques

Abrégé

The present invention relates to administration of gene therapy, in particular the development of effective combinatorial strategies for gene delivery in inter alia monogenetic diseases. More specifically, the present invention relates to a combination therapy, methods of producing the combination therapy, as well as various related embodiments.

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C12N 15/86 - Vecteurs viraux

Produits et services

Pharmaceutical preparations and substances for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes in the form of a liquid solution or lyophilised preparation for therapeutic use for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; pharmaceutical preparations, namely, exosomes to deliver protein or nucleic acids or viral vectors for genetic therapy; medicine for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; Medical preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations for medical purposes for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; biological preparations comprising exosome for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations comprising a therapeutic target protein-loaded, nucleic acid-loaded or virus-loaded exosome for the treatment of genetic diseases, respiratory diseases, or cancer; biological reagents for medical use; biological preparations for medical purposes, namely, for the prevention and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; mixed biological preparations for the prevention and treatment of infectious diseases; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, EV-based nucleic acid therapeutic agents for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; scientific research and analysis services; biological research; clinical research in the field of transformational therapeutics; providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood; scientific investigations for medical purposes; scientific laboratory services; medical laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; information, advisory and consultancy services relating to the aforesaid, namely, scientific, medical and pharmaceutical research and development services, pharmaceutical drug development services, research and development of pharmaceutical preparations, scientific research and analysis services, biological research, clinical research in the field of transformational therapeutics, providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood, scientific investigations for medical purposes, scientific laboratory services, medical laboratory services, laboratory research services relating to pharmaceuticals, research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders, biotechnology, pharmaceutical research and development and genetic science, laboratory diagnostics, namely, medical analysis services relating to the diagnosis and treatment of persons provided by medical laboratories Medical services; medical and pharmaceutical consultancy; medical diagnostics, namely, medical testing, monitoring, and reporting services for diagnostic or treatment purposes; medical analysis services for the diagnosis and treatment of persons and for the diagnosis and treatment of genetic diseases; medical examination services; medical treatment services in the field of biotherapeutics for the treatment of infectious diseases, cancer, neurological disorders, neurodegenerative disorders, inflammatory disorders, rare diseases, genetic and metabolic disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; information, advisory and consultancy services relating to the aforesaid, namely, medical services, medical and pharmaceutical consultancy, medical diagnostics, namely, medical testing, monitoring, and reporting services, medical analysis services for the diagnosis and treatment of persons and for the diagnosis and treatment of genetic diseases, medical examination services, medical treatment services in the field of biotherapeutics for the treatment of infectious diseases, cancer, neurological disorders, neurodegenerative disorders, inflammatory disorders, rare diseases, genetic and metabolic disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases, medical screening, medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases, and medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears and ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders

Produits et services

Pharmaceutical preparations and substances for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes in the form of a liquid solution or lyophilised preparation for therapeutic use for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; pharmaceutical preparations, namely, exosomes to deliver protein or nucleic acids or viral vectors for genetic therapy; medicine for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; Medical preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations for medical purposes for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; biological preparations comprising exosome for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations comprising a therapeutic target protein-loaded, nucleic acid-loaded or virus-loaded exosome for the treatment of genetic diseases, respiratory diseases, or cancer; biological reagents for medical use; biological preparations for medical purposes, namely, for the prevention and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; mixed biological preparations for the prevention and treatment of infectious diseases; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, EV-based nucleic acid therapeutic agents for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; scientific research and analysis services; biological research; clinical research in the field of transformational therapeutics; providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood; scientific investigations for medical purposes; scientific laboratory services; medical laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; information, advisory and consultancy services relating to the aforesaid, namely, scientific, medical and pharmaceutical research and development services, pharmaceutical drug development services, research and development of pharmaceutical preparations, scientific research and analysis services, biological research, clinical research in the field of transformational therapeutics, providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood, scientific investigations for medical purposes, scientific laboratory services, medical laboratory services, laboratory research services relating to pharmaceuticals, research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders, biotechnology, pharmaceutical research and development and genetic science, laboratory diagnostics, namely, medical analysis services relating to the diagnosis and treatment of persons provided by medical laboratories Medical services; medical and pharmaceutical consultancy; medical diagnostics, namely, medical testing, monitoring, and reporting services for diagnostic or treatment purposes; medical analysis services for the diagnosis and treatment of persons and for the diagnosis and treatment of genetic diseases; medical examination services; medical treatment services in the field of biotherapeutics for the treatment of infectious diseases, cancer, neurological disorders, neurodegenerative disorders, inflammatory disorders, rare diseases, genetic and metabolic disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; information, advisory and consultancy services relating to the aforesaid, namely, medical services, medical and pharmaceutical consultancy, medical diagnostics, namely, medical testing, monitoring, and reporting services, medical analysis services for the diagnosis and treatment of persons and for the diagnosis and treatment of genetic diseases, medical examination services, medical treatment services in the field of biotherapeutics for the treatment of infectious diseases, cancer, neurological disorders, neurodegenerative disorders, inflammatory disorders, rare diseases, genetic and metabolic disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases, medical screening, medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases, and medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears and ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders

Produits et services

Pharmaceutical preparations and substances for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes in the form of a liquid solution or lyophilised preparation for therapeutic use for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; pharmaceutical preparations, namely, exosomes to deliver protein or nucleic acids or viral vectors for genetic therapy; medicine for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; Medical preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations for medical purposes for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; biological preparations comprising exosome for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; biological preparations comprising a therapeutic target protein-loaded, nucleic acid-loaded or virus-loaded exosome for the treatment of genetic diseases, respiratory diseases, or cancer; biological reagents for medical use; biological preparations for medical purposes, namely, for the prevention and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders, urea cycle disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; mixed biological preparations for the prevention and treatment of infectious diseases; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, EV-based nucleic acid therapeutic agents for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; scientific research and analysis services; biological research; clinical research in the field of transformational therapeutics; providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood; scientific investigations for medical purposes; scientific laboratory services; medical laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; information, advisory and consultancy services relating to the aforesaid, namely, scientific, medical and pharmaceutical research and development services, pharmaceutical drug development services, research and development of pharmaceutical preparations, scientific research and analysis services, biological research, clinical research in the field of transformational therapeutics, providing medical and scientific research information in the field of filtration of exosomes from cultured cells or blood, scientific investigations for medical purposes, scientific laboratory services, medical laboratory services, laboratory research services relating to pharmaceuticals, research and development in the field of biotherapeutics for the treatment of genetic diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders, biotechnology, pharmaceutical research and development and genetic science, laboratory diagnostics, namely, medical analysis services relating to the diagnosis and treatment of persons provided by medical laboratories Medical services; medical and pharmaceutical consultancy; medical diagnostics, namely, medical testing, monitoring, and reporting services for diagnostic or treatment purposes; medical analysis services for the diagnosis and treatment of persons and for the diagnosis and treatment of genetic diseases; medical examination services; medical treatment services in the field of biotherapeutics for the treatment of infectious diseases, cancer, neurological disorders, neurodegenerative disorders, inflammatory disorders, rare diseases, genetic and metabolic disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders; information, advisory and consultancy services relating to the aforesaid, namely, medical services, medical and pharmaceutical consultancy, medical diagnostics, namely, medical testing, monitoring, and reporting services, medical analysis services for the diagnosis and treatment of persons and for the diagnosis and treatment of genetic diseases, medical examination services, medical treatment services in the field of biotherapeutics for the treatment of infectious diseases, cancer, neurological disorders, neurodegenerative disorders, inflammatory disorders, rare diseases, genetic and metabolic disorders, liver disorders and diseases, brain diseases, eye disorders and diseases and lung disorders and diseases, medical screening, medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, viral and bacterial based infections and diseases, and medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies being mitochondrial disease, joint strain and tears and ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism (IEMs), lysosomal storage disorders and urea cycle disorders

Abrégé

The present invention pertains to affinity chromatography isolation and purification of extracellular vesicles (EVs). The EVs of the present invention are engineered to enable highly specific binding to e.g. chromatography matrices, which is highly useful for affinity-based isolation and purification of EVs from complex biological fluids such as cell culture medium or biological fluids.

Classes IPC  ?

• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
• A61K 35/55 - Glandes non prévues dans les groupes , p. ex. glandes thyroïdes, parathyroïdes ou pinéales
• B01D 15/38 - Adsorption sélective, p. ex. chromatographie caractérisée par le mécanisme de séparation impliquant une interaction spécifique non couverte par un ou plusieurs des groupes , p. ex. chromatographie d'affinité, chromatographie d'échange par ligand ou chromatographie chirale
• C07K 14/70 - Enképhalines
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• C07K 14/71 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des facteurs de croissanceRécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des régulateurs de croissance
• C07K 14/715 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des cytokinesRécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des lymphokinesRécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des interférons

Abrégé

The present invention pertains to a new nanoparticle-like delivery system for intracellular delivery of cargo molecules such as nucleic acids, ribonucleoproteins and extracellular vesicles.

Classes IPC  ?

• C07K 7/08 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 12 à 20 amino-acides
• A61K 38/04 - Peptides ayant jusqu'à 20 amino-acides dans une séquence entièrement déterminéeLeurs dérivés
• C07K 7/06 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 5 à 11 amino-acides

Abrégé

The present invention pertains to a new nanoparticle-like delivery system for intracellular delivery of cargo molecules such as nucleic acids, ribonucleoproteins and extracellular vesicles.

Classes IPC  ?

• C07K 7/08 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 12 à 20 amino-acides
• C07K 7/06 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 5 à 11 amino-acides
• A61K 38/04 - Peptides ayant jusqu'à 20 amino-acides dans une séquence entièrement déterminéeLeurs dérivés

Abrégé

The present invention pertains to extracellular vesicle (EV) therapeutics, wherein the EVs comprise nucleic acid (NA)-based therapeutics such as mRNAs, circular RNAs, miRNAs, shRNAs, and/or DNA molecules. The NA therapeutics are loaded into EVs using inventive protein and NA engineering strategies which stabilize the cargo NAs and enhance loading into EVs, thereby enhancing therapeutic activity of the cargo NA molecules half-life.

Classes IPC  ?

• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
• C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire

Abrégé

The present invention pertains to extracellular vesicle (EV) therapeutics, wherein the EVs comprise nucleic acid (NA)-based therapeutics such as mRNAs, circular RNAs, miRNAs, shRNAs, and/or DNA molecules. The NA therapeutics are loaded into EVs using inventive protein and NA engineering strategies which stabilize the cargo NAs and enhance loading into EVs, thereby enhancing therapeutic activity of the cargo NA molecules half-life.

Classes IPC  ?

• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
• C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques

Abrégé

The present disclosure relates to extracellular vesicles, e.g., exosomes, comprising an AAV and a scaffold protein. In some aspects, the AAV is in the lumen of the extracellular vesicle. In some aspects, the AAV is associated with the luminal surface of the extracellular vesicle. In some aspects, the AAV is associated with the exterior surface of the extracellular vesicle. Also provided herein are methods for producing the exosomes and methods for using the exosomes to treat and/or prevent diseases or disorders.

Classes IPC  ?

• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• B82Y 5/00 - Nanobiotechnologie ou nanomédecine, p. ex. génie protéique ou administration de médicaments
• A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique
• A61K 9/133 - Vésicules unilamellaires
• A61K 9/51 - Nanocapsules
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C07K 14/005 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant de virus
• C07K 14/015 - Parvoviridae, p. ex. virus de l'aleucémie féline, parvovirus humain
• C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
• C12N 5/10 - Cellules modifiées par l'introduction de matériel génétique étranger, p. ex. cellules transformées par des virus
• C12N 15/864 - Vecteurs parvoviraux

Produits et services

Chemical reagents; Pharmaceuticals; pharmaceutical preparations and substances; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein for genetic therapy; medicine; medical preparations and articles; biological preparations for medical purposes; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy; biological preparations comprising exosome; biological preparations comprising a therapeutic target protein-loaded exosome; biological preparations comprising exosome for pharmaceutical purposes; biological reagents for medical use; mixed biological preparations for medical purposes; mixed biological preparations for the prevention and treatment of infectious diseases; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, EV-based nucleic acid therapeutics for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy; none of the aforesaid goods being exclusively for use in relation to the development and production of industrial enzymes or probiotics used in food and drinks applications. Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; research and analysis services; biological research; clinical research; providing medical and scientific research information in the field of filtration of exosomes from blood; scientific investigations for medical purposes; scientific laboratory services; medical research laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of infectious diseases and metabolic disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science; information, advisory and consultancy services in relation to the aforesaid; none of the aforesaid services being exclusively for use in relation to the development and production of industrial enzymes or probiotics used in food and drinks applications. Medical services; medical and pharmaceutical consultancy; medical services relating to the diagnosis and treatment of persons; laboratory analysis service relating to the diagnosis and treatment of persons; medical analysis and examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy; information, advisory and consultancy services in relation to the aforesaid.

Abrégé

The present invention pertains to inter alia methods for purifying extracellular vesicles including exposing a sample comprising at least one extracellular vesicle to ultrafiltration; and exposing the sample following the ultrafiltration in step (i) to size exclusion liquid chromatography.

Classes IPC  ?

• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 47/62 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant une protéine, un peptide ou un acide polyaminé
• A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
• A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
• A61K 38/00 - Préparations médicinales contenant des peptides
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• F01C 21/10 - Organes externes coopérant avec des pistons rotatifsCarcasses d'enveloppes
• F01C 1/344 - "Machines" ou machines motrices à piston rotatif ayant des caractéristiques couvertes par au moins deux des groupes , , , ou par l'un de ces groupes associées à un autre type de mouvement entre les organes coopérants ayant à la fois le mouvement défini dans le groupe ou et un mouvement alternatif entre les organes coopérants comportant des organes obturateurs se déplaçant par rapport à l'organe interne

Abrégé

The present invention relates to engineered extracellular vesicles (EVs) as a novel therapeutic approach to treating urea cycle disorders. More specifically, the invention relates to the use of various protein engineering and nucleic acid engineering strategies for improving loading of urea cycle proteins or nucleic acids encoding urea cycle proteins into EVs and targeting of the resultant EVs to tissues and organs of interest.

Classes IPC  ?

• C12N 9/88 - Lyases (4.)
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit

Abrégé

The present invention relates to engineered extracellular vesicles (EVs) as a novel therapeutic approach to treating urea cycle disorders. More specifically, the invention relates to the use of various protein engineering and nucleic acid engineering strategies for improving loading of urea cycle proteins or nucleic acids encoding urea cycle proteins into EVs and targeting of the resultant EVs to tissues and organs of interest.

Classes IPC  ?

• C12N 9/88 - Lyases (4.)
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit

Abrégé

The present invention pertains to extracellular vesicle (EV) therapeutics, wherein the EVs are coated with proteins containing Fc domains (such as antibodies) for i.a. targeting and therapeutic applications. The coating of EVs is achieved through inventive protein engineering of EV polypeptides. The present invention thus relates to methods for coating of EVs, EVs per se, as well as pharmaceutical compositions and medical applications of such EVs coated with Fc containing proteins.

Classes IPC  ?

• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique
• A61K 47/24 - Composés organiques, p. ex. hydrocarbures naturels ou synthétiques, polyoléfines, huile minérale, gelée de pétrole ou ozocérite contenant des atomes autres que des atomes de carbone, d'hydrogène, d'oxygène, d'halogènes, d'azote ou de soufre, p. ex. cyclométhicone ou phospholipides
• C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
• A61K 38/00 - Préparations médicinales contenant des peptides

Produits et services

Pharmaceuticals; pharmaceutical preparations and substances; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein for genetic therapy; medicine; medical preparations and articles; biological preparations for medical purposes; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy; biological preparations comprising exosome; biological preparations comprising a therapeutic target protein-loaded exosome; biological preparations comprising exosome for pharmaceutical purposes; biological reagents for medical use; mixed biological preparations for medical purposes; mixed biological preparations for the prevention and treatment of infectious diseases; diagnostic reagents used in the fields of biological, genetic engineering and pharmaceutical research; diagnostic reagents comprising exosome used in the fields of biological, genetic engineering and pharmaceutical research; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, EV-based nucleic acid therapeutics for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy. Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; research and analysis services; biological research; clinical research; providing medical and scientific research information in the field of filtration of exosomes from blood; scientific investigations for medical purposes; scientific laboratory services; medical research laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of infectious diseases and metabolic disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science. Medical services; medical and pharmaceutical consultancy; medical services relating to the diagnosis and treatment of persons; laboratory analysis service relating to the diagnosis and treatment of persons; medical analysis and examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy.

Produits et services

Pharmaceutical preparations and substances for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies in the nature of mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism, lysosomal storage disorders and urea cycle disorders; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered and purified exosomes in the form of a liquid solution and lyophilised preparation for therapeutic use in the nature of treatment of for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies in the nature of mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism, lysosomal storage disorders and urea cycle disorders; pharmaceutical preparations, namely, exosomes to deliver protein for genetic therapy; Medicine for treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies in the nature of mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism, lysosomal storage disorders and urea cycle disorders; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint and ligament strains and tears, muscle atrophy, muscular dystrophy; mixed biological preparations for the prevention and treatment of infectious diseases; EV-based nucleic acid therapeutics in the nature of pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars, and therapeutics for the prevention and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies in the nature of mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, genetic diseases, rare diseases, in-born errors of metabolism, lysosomal storage disorders and urea cycle disorders; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies in the nature of mitochondrial disease, joint strains and tears, ligament strains and tears, muscle atrophy, and muscular dystrophy Medical services; medical and pharmaceutical consultancy; medical services relating to the diagnosis and treatment of persons; medical analysis service relating to the diagnosis and treatment of persons provided by medical laboratories; medical analysis and examinations for the diagnosis of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies in the nature of mitochondrial disease, joint strain and tears, ligament strain and tears, muscle atrophy, muscular dystrophy, neuromuscular diseases, neurological disorders, neurodegenerative disorders, inflammatory disorders, infectious diseases, rare diseases, genetic diseases, in-born errors of metabolism, lysosomal storage disorders and urea cycle disorders; medical treatment services in the field of biotherapeutics for the treatment of infectious diseases, cancer, neurological disorders, neurodegenerative disorders, inflammatory disorders, rare diseases, genetic and metabolic disorders; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies in the nature of mitochondrial disease, joint and ligament strains and tears, muscle atrophy, muscular dystrophy

Abrégé

The present invention relates to administration of gene therapy, in particular the development of effective combinatorial strategies for gene delivery in inter alia monogenetic diseases. More specifically, the present invention relates to a combination therapy, methods of producing the combination therapy, as well as various related embodiments.

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique

Abrégé

The present invention relates to methods for loading extracellular vesicles (EVs) with a pharmacological agent. The invention discloses the use of cell-penetrating peptides as carriers into EVs, using either a non-covalent or covalent loading approach. Furthermore, the present invention pertains to medical uses and compositions comprising such pharmacological agent-loaded EVs.

Classes IPC  ?

• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 9/127 - Vecteurs à bicouches synthétiques, p. ex. liposomes ou liposomes comportant du cholestérol en tant qu’unique agent tensioactif non phosphatidylique
• A61K 47/61 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique macromoléculaire, p. ex. une molécule oligomérique, polymérique ou dendrimérique le composé organique macromoléculaire étant un polysaccharide ou l’un de ses dérivés
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
• A61K 9/50 - Microcapsules
• C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques

Abrégé

The present invention relates to administration of gene therapy, in particular the development of effective combinatorial strategies for gene delivery in inter alia monogenetic diseases. More specifically, the present invention relates to a combination therapy, methods of producing the combination therapy, as well as various related embodiments.

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique

Abrégé

The present invention pertains to affinity-based isolation and purification of drug- loaded extracellular vesicles, notably exosomes, wherein the exosomes are engineered to enable affinity purification.

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• C12N 15/88 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation utilisant la micro-encapsulation, p. ex. utilisant des vésicules liposomiques
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif

Produits et services

Pharmaceuticals; pharmaceutical preparations and substances; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein for genetic therapy; medicine; medical preparations and articles; biological preparations for medical purposes; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy; biological preparations comprising exosome; biological preparations comprising a therapeutic target protein-loaded exosome; biological preparations comprising exosome for pharmaceutical purposes; biological reagents for medical use; mixed biological preparations for medical purposes; mixed biological preparations for the prevention and treatment of infectious diseases; diagnostic reagents used in the fields of biological, genetic engineering and pharmaceutical research; diagnostic reagents comprising exosome used in the fields of biological, genetic engineering and pharmaceutical research; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, EV-based nucleic acid therapeutics for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy. Medical services; medical and pharmaceutical consultancy; medical services relating to the diagnosis and treatment of persons; laboratory analysis service relating to the diagnosis and treatment of persons; medical analysis and examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy.

Abrégé

The present invention relates to engineered cells that enable exosome-mediated delivery of therapeutic cargoes, in particular protein therapeutics and RNA therapeutics. The present invention also relates to inventive polynucleotides, polypeptides and pharmaceutical compositions.

Classes IPC  ?

• C07K 19/00 - Peptides hybrides
• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire

Abrégé

The present invention relates to compositions and buffers for storing engineered extracellular vesicles (EVs), such as exosomes, for extended time periods with maintained stability and function. The present invention allows for both optimized long-term storage and for clinical application of the stored EVs.

Classes IPC  ?

• A61K 47/02 - Composés inorganiques
• A61K 47/26 - Hydrates de carbone, p. ex. polyols ou sucres alcoolisés, sucres aminés, acides nucléiques, mono-, di- ou oligosaccharidesLeurs dérivés, p. ex. polysorbates, esters d’acide gras de sorbitan ou glycyrrhizine
• A61K 47/42 - ProtéinesPolypeptidesLeurs produits de dégradationLeurs dérivés p. ex. albumine, gélatine ou zéine
• A61K 35/12 - Substances provenant de mammifèresCompositions comprenant des tissus ou des cellules non spécifiésCompositions comprenant des cellules souches non embryonnairesCellules génétiquement modifiées
• A61P 29/00 - Agents analgésiques, antipyrétiques ou anti-inflammatoires non centraux, p. ex. agents antirhumatismauxMédicaments anti-inflammatoires non stéroïdiens [AINS]
• A61K 47/10 - AlcoolsPhénolsLeurs sels, p. ex. glycérolPolyéthylène glycols [PEG]PoloxamèresAlkyléthers de PEG/POE
• A61K 47/18 - AminesAmidesUréesComposés d’ammonium quaternaireAcides aminésOligopeptides ayant jusqu’à cinq acides aminés
• A61K 47/20 - Composés organiques, p. ex. hydrocarbures naturels ou synthétiques, polyoléfines, huile minérale, gelée de pétrole ou ozocérite contenant du soufre, p. ex. sulfoxyde de diméthyle [DMSO], docusate, laurylsulfate de sodium ou acides aminosulfoniques

Abrégé

The present invention relates to extracellular vesicle (EV)-mediated delivery of protein- based therapeutics. More specifically, the invention relates to delivery of complex polypeptide-based agents which typically bind to target proteins extracellularly, intracellularly, or in the cell membrane.

Classes IPC  ?

• A61K 38/53 - Ligases (6)
• C12N 9/00 - Enzymes, p. ex. ligases (6.)ProenzymesCompositions les contenantProcédés pour préparer, activer, inhiber, séparer ou purifier des enzymes
• A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire

Abrégé

The present invention pertains to extracellular vesicle (EV) therapeutics, wherein the EVs comprise nucleic acid (NA)-based therapeutics such as m RNAs, circular RNAs, mi RNAs, sh RNAs, circular RNA and/or DNA molecules. The NA therapeutics are loaded into EVs using inventive engineering protein and NA engineering strategies to enhance loading into EVs and to facilitate release of the NA cargo molecules inside target cells.

Classes IPC  ?

• A61K 41/00 - Préparations médicinales obtenues par traitement de substances par énergie ondulatoire ou par rayonnement corpusculaire
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• A61K 47/65 - Séquences de liaison, liants ou bras-espaceurs peptidiques, p. ex. séquences de liaison peptidiques vulnérable aux protéases

Abrégé

The present invention relates to extracellular vesicles (EVs) as a novel therapeutic approach to lysosomal storage disorders (LSD). More specifically, the invention relates to the use of various protein engineering strategies for improving loading of hard-to-load LSD-related proteins and targeting of the resultant EVs to tissues and organs of interest.

Classes IPC  ?

• A61K 35/50 - PlacentaCellules souches placentairesLiquide amniotiqueAmniosCellules souches amniotiques
• C12N 5/073 - Cellules ou tissus embryonnairesCellules fœtales ou tissus fœtaux
• A61K 9/50 - Microcapsules
• A61K 38/00 - Préparations médicinales contenant des peptides
• A61P 3/00 - Médicaments pour le traitement des troubles du métabolisme

Abrégé

The present invention pertains to extracellular vesicle (EV) therapeutics, wherein the EVs comprise nucleic acid (NA)-based therapeutics such as m RNAs, circular RNAs, mi RNAs, sh RNAs, circular RNA and/or DNA molecules. The NA therapeutics are loaded into EVs using inventive engineering protein and NA engineering strategies to enhance loading into EVs and to facilitate release of the NA cargo molecules inside target cells.

Classes IPC  ?

• A61K 41/00 - Préparations médicinales obtenues par traitement de substances par énergie ondulatoire ou par rayonnement corpusculaire
• A61K 47/65 - Séquences de liaison, liants ou bras-espaceurs peptidiques, p. ex. séquences de liaison peptidiques vulnérable aux protéases
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif

Abrégé

The present invention relates to extracellular vesicles (EVs) as a novel therapeutic approach to lysosomal storage disorders (LSD). More specifically, the invention relates to the use of various protein engineering strategies for improving loading of hard-to-load LSD-related proteins and targeting of the resultant EVs to tissues and organs of interest.

Classes IPC  ?

• A61K 35/50 - PlacentaCellules souches placentairesLiquide amniotiqueAmniosCellules souches amniotiques
• A61K 38/00 - Préparations médicinales contenant des peptides
• A61P 3/00 - Médicaments pour le traitement des troubles du métabolisme
• A61K 9/50 - Microcapsules
• C12N 5/073 - Cellules ou tissus embryonnairesCellules fœtales ou tissus fœtaux

Abrégé

The present invention pertains to affinity chromatography isolation and purification of extracellular vesicles (EVs). The EVs of the present invention are engineered to enable highly specific binding to e.g. chromatography matrices, which is highly useful for affinity-based isolation and purification of EVs from complex biological fluids such as cell culture medium or biological fluids.

Classes IPC  ?

• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
• A61K 47/06 - Composés organiques, p. ex. hydrocarbures naturels ou synthétiques, polyoléfines, huile minérale, gelée de pétrole ou ozocérite

Abrégé

The present invention pertains to improved methods for loading of extracellular vesicles (EVs), such as exosomes, with various types of proteins of interest. More specifically, the invention relates to loading of EVs using fusion polypeptide constructs, as well as i.a. fusion constructs perse and EVs carrying such fusion polypeptides. The design of the fusion polypeptides is key to enable both efficient surface-display and internal loading into EVs of proteins of interest.

Classes IPC  ?

• C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères

Abrégé

The present invention pertains to engineered extracellular vesicles(EVs) for use in a variety of research, diagnostic, imaging and therapeutic applications. The EVs are engineered to enable incorporation and surface display of various proteins of interest, which may be exogenous and/or endogenous in nature. The coating of EVs is achieved through inventive protein engineering of EV polypeptides, and the present invention thus relates to methods for coating of EVs, EVs per se, as well kits, detection methods, diagnostic applications, imaging and delivery methods based on said engineered EVs.

Classes IPC  ?

• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire

Abrégé

The present invention pertains to extracellular vesicle (EV) therapeutics, wherein the EVs are coated with proteins containing Fc domains (such as antibodies) for i.a. targeting and therapeutic applications. The coating of EVs is achieved through inventive protein engineering of EV polypeptides. The present invention thus relates to methods for coating of EVs, EVs per se, as well as pharmaceutical compositions and medical applications of such EVs coated with Fc containing proteins.

Classes IPC  ?

• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire

Abrégé

The present invention pertains to extracellular vesicle (EV) therapeutics, wherein the EVs are coated with proteins containing Fc domains (such as antibodies) for i.a. targeting and therapeutic applications. The coating of EVs is achieved through inventive protein engineering of EV polypeptides. The present invention thus relates to methods for coating of EVs, EVs per se, as well as pharmaceutical compositions and medical applications of such EVs coated with Fc containing proteins.

Classes IPC  ?

• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire

Abrégé

The present invention pertains to engineered extracellular vesicles(EVs) for use in a variety of research, diagnostic, imaging and therapeutic applications. The EVs are engineered to enable incorporation and surface display of various proteins of interest, which may be exogenous and/or endogenous in nature. The coating of EVs is achieved through inventive protein engineering of EV polypeptides, and the present invention thus relates to methods for coating of EVs, EVs per se, as well kits, detection methods, diagnostic applications, imaging and delivery methods based on said engineered EVs.

Classes IPC  ?

• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire

Abrégé

The present invention relates to methods for metabolically loading extracellular vesicles (EVs) with a pharmacological agent. The methods comprise culturing EV source cells in the presence of a metabolic component comprising a pharmacological agent, which is thereby incorporated into the EV-producing cells and subsequently into the EV. The invention further relates to medical uses and compositions of such EVs.

Classes IPC  ?

• A61K 9/14 - Préparations médicinales caractérisées par un aspect particulier à l'état particulaire, p. ex. poudres
• C12N 5/071 - Cellules ou tissus de vertébrés, p. ex. cellules humaines ou tissus humains
• A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
• A61K 47/66 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant une protéine, un peptide ou un acide polyaminé l’agent de modification étant un système de pré-ciblage impliquant un peptide ou une protéine pour cibler des cellules spécifiques
• A61K 47/68 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un anticorps, une immunoglobuline ou son fragment, p. ex. un fragment Fc
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 9/51 - Nanocapsules

Abrégé

The present invention relates to methods for metabolically loading extracellular vesicles (EVs) with a pharmacological agent. The methods comprise culturing EV source cells in the presence of a metabolic component comprising a pharmacological agent, which is thereby incorporated into the EV-producing cells and subsequently into the EV. The invention further relates to medical uses and compositions of such EVs.

Classes IPC  ?

• A61K 9/48 - Préparations en capsules, p. ex. de gélatine, de chocolat
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61K 35/13 - Cellules tumorales, quel que soit le tissu d’origine
• A61K 35/545 - Cellules souches embryonnairesCellules souches pluripotentesCellules souches pluripotentes induitesCellules souches non caractérisées
• A61K 47/66 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant une protéine, un peptide ou un acide polyaminé l’agent de modification étant un système de pré-ciblage impliquant un peptide ou une protéine pour cibler des cellules spécifiques

Abrégé

The present invention relates to methods for loading extracellular vesicles (EVs) with a pharmacological agent. The invention discloses the use of cell-penetrating peptides as carriers into EVs, using either a non-covalent or covalent loading approach. Furthermore, the present invention pertains to medical uses and compositions comprising such pharmacological agent-loaded EVs.

Classes IPC  ?

• A61K 47/50 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament

Abrégé

The present invention relates to extracellular vesicles (EVs) comprising a binding protein which may be used for delivery of protein-drug conjugates comprising the binding protein and a small molecule agent, typically a small molecule drug. The present invention also relates to methods for producing such EVs as well as pharmaceutical compositions and medical uses of such EVs.

Classes IPC  ?

• A61K 47/50 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament

Abrégé

The present invention pertains to an inventive release mechanism for extracellular vesicle (EV)-mediated intracellular and intramembrane delivery of therapeutic polypeptides. More specifically, the invention relates to EVs comprising polypeptide constructs which comprise a therapeutic polypeptide releasably attached to an exosomal polypeptide. Furthermore, the present invention pertains to manufacturing methods, pharmaceutical compositions, medical uses and applications, and various other embodiments related to the inventive EVs.

Classes IPC  ?

• C12N 15/62 - Séquences d'ADN codant pour des protéines de fusion
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• C07K 17/04 - Peptides immobilisés sur, ou dans, un support organique piégés à l'intérieur du support, p. ex. dans un gel, dans une fibre creuse
• C07K 19/00 - Peptides hybrides
• C12N 5/10 - Cellules modifiées par l'introduction de matériel génétique étranger, p. ex. cellules transformées par des virus
• C12N 15/87 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation
• C12P 21/00 - Préparation de peptides ou de protéines

Abrégé

The present invention pertains to an inventive release mechanism for extracellular vesicle (EV)-mediated intracellular and intramembrane delivery of therapeutic polypeptides. More specifically, the invention relates to EVs comprising polypeptide constructs which comprise a therapeutic polypeptide releasably attached to an exosomal polypeptide. Furthermore, the present invention pertains to manufacturing methods, pharmaceutical compositions, medical uses and applications, and various other embodiments related to the inventive EVs.

Classes IPC  ?

• A61K 41/00 - Préparations médicinales obtenues par traitement de substances par énergie ondulatoire ou par rayonnement corpusculaire
• A61K 47/65 - Séquences de liaison, liants ou bras-espaceurs peptidiques, p. ex. séquences de liaison peptidiques vulnérable aux protéases
• A61K 47/64 - Conjugués médicament-peptide, médicament-protéine ou médicament-acide polyaminé, c.-à-d. l’agent de modification étant un peptide, une protéine ou un acide polyaminé lié par covalence ou complexé à un agent thérapeutiquement actif

Produits et services

Pharmaceuticals; pharmaceutical preparations and substances; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein for genetic therapy; medicine; medical preparations and articles; biological preparations for medical purposes; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy; biological preparations comprising exosome for pharmaceutical purposes; biological preparations comprising a therapeutic target protein-loaded exosome; biological preparations comprising exosome for use in the manufacture of pharmaceuticals; biological reagents for medical use; mixed biological preparations for medical purposes; mixed biological preparations for the prevention and treatment of infectious diseases; diagnostic reagents used in the fields of biological, genetic engineering and pharmaceutical research; diagnostic reagents comprising exosome used in the fields of biological, genetic engineering and pharmaceutical research; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, ev-based nucleic acid therapeutics for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy. Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; research and analysis services; biological research; clinical research; providing medical and scientific research information in the field of filtration of exosomes from blood; scientific investigations for medical purposes; scientific laboratory services; medical research laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of infectious diseases and metabolic disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science. Medical services; medical and pharmaceutical consultancy; medical services relating to diagnosis and treatment for persons; laboratory analysis services relating to the diagnosis and treatment of persons; medical analysis and examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy.

Produits et services

Pharmaceuticals; pharmaceutical preparations and substances; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for therapeutic use; pharmaceutical preparations, namely, exosomes to deliver protein for genetic therapy; medicine; medical preparations and articles; biological preparations for medical purposes; biological preparations for the treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy; biological preparations comprising exosome; biological preparations comprising a therapeutic target protein-loaded exosome; biological preparations comprising exosome for pharmaceutical purposes; biological reagents for medical use; mixed biological preparations for medical purposes; mixed biological preparations for the prevention and treatment of infectious diseases; diagnostic reagents used in the fields of biological, genetic engineering and pharmaceutical research; diagnostic reagents comprising exosome used in the fields of biological, genetic engineering and pharmaceutical research; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, EV-based nucleic acid therapeutics for the prevention and treatment of infections or disease; diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in disease detection; diagnostic kits comprised of medical diagnostic reagents and assays that test for the presence of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy. Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; research and analysis services; biological research; clinical research; providing medical and scientific research information in the field of filtration of exosomes from blood; scientific investigations for medical purposes; scientific laboratory services; medical research laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of infectious diseases and metabolic disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science. Medical services; medical and pharmaceutical consultancy; medical services relating to the diagnosis and treatment of persons; laboratory analysis service relating to the diagnosis and treatment of persons; medical analysis and examinations; medical treatment services; medical screening; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of viral and bacterial based infections and diseases; medical monitoring, testing, inspection and analysis services relating to the diagnosis and treatment of cancer, diabetes, obesity, metabolic syndrome, dementia, and other aging associated pathologies such as mitochondrial disease, joint/ligament strain/tears, muscle atrophy, muscular dystrophy.

Produits et services

Drug delivery agents in the form of exosomes that facilitate the delivery of pharmaceutical preparations; pharmaceutical preparations, namely, a drug delivery system comprising genetically engineered or purified exosomes for transport of therapeutic agents; pharmaceutical preparations, namely, engineered exosomes that deliver protein for genetic therapy; biological preparations comprising exosome for medical use; biological reagents comprising exosome for medical use; mixed biological preparations comprising exosome for pharmaceutical and medical use; diagnostic reagents comprising exosome for medicinal use in the fields of biology, genetic engineering and pharmaceuticals; pharmaceuticals, medicines, biologics, biotherapeutics, biosimilars and therapeutics, namely, extracellular vesicle-based nucleic acid therapeutics for the prevention and treatment of infectious diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, mitochondrial disease, neuromuscular disease, inflammatory diseases and autoimmune disorders; diagnostic kits comprised of medical diagnostic reagents and assays for testing of exosomes for use in the detection of infectious diseases, cancer, diabetes, obesity, metabolic syndrome, dementia, mitochondrial disease, neuromuscular disease, inflammatory diseases and autoimmune disorders Scientific, medical and pharmaceutical research and development services; pharmaceutical drug development services; research and development of pharmaceutical preparations; biological research; clinical research in the field of transformational therapeutics; providing medical and scientific research information in the field of filtration of exosomes from blood; scientific investigations for medical purposes; scientific laboratory services; medical research laboratory services; laboratory research services relating to pharmaceuticals; research and development in the field of biotherapeutics for the treatment of infectious diseases and metabolic disorders; consulting services in the fields of biotechnology, pharmaceutical research and development and genetic science

Abrégé

The present invention pertains to inter alia therapeutic delivery vesicles, for instance exosomes or microvesicles, comprising polypeptide constructs, methods for producing said therapeutic delivery vesicles, pharmaceutical compositions and medical uses thereof. The therapeutic polypeptide constructs comprised in the extracellular delivery vesicles enable sequestering target molecules of interest, to treat e.g. neuro-inflammatory diseases and cancer.

Classes IPC  ?

• A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
• A61K 38/00 - Préparations médicinales contenant des peptides
• C07K 2/00 - Peptides à nombre indéterminé d'amino-acidesLeurs dérivés
• C07K 4/00 - Peptides ayant jusqu'à 20 amino-acides dans une séquence indéterminée ou partiellement déterminéeLeurs dérivés
• C07K 5/00 - Peptides ayant jusqu'à quatre amino-acides dans une séquence entièrement déterminéeLeurs dérivés
• C07K 7/00 - Peptides ayant de 5 à 20 amino-acides dans une séquence entièrement déterminéeLeurs dérivés
• C07K 14/00 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés
• C07K 16/00 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux
• C07K 17/00 - Peptides fixés sur un support ou immobilisésLeur préparation
• C07K 1/00 - Procédés généraux de préparation de peptides
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• F01C 21/10 - Organes externes coopérant avec des pistons rotatifsCarcasses d'enveloppes
• F01C 1/344 - "Machines" ou machines motrices à piston rotatif ayant des caractéristiques couvertes par au moins deux des groupes , , , ou par l'un de ces groupes associées à un autre type de mouvement entre les organes coopérants ayant à la fois le mouvement défini dans le groupe ou et un mouvement alternatif entre les organes coopérants comportant des organes obturateurs se déplaçant par rapport à l'organe interne
• A61K 47/69 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament le conjugué étant caractérisé par sa forme physique ou sa forme galénique, p. ex. émulsion, particule, complexe d’inclusion, stent ou kit
• A61K 47/62 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant une protéine, un peptide ou un acide polyaminé
• A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains

Abrégé

The present invention pertains to inter alia therapeutic delivery vesicles, for instance exosomes or microvesicles, comprising polypeptide constructs, methods for producing said therapeutic delivery vesicles, pharmaceutical compositions and medical uses thereof. The therapeutic polypeptide constructs comprised in the extracellular delivery vesicles enable sequestering target molecules of interest, to treat e.g. neuro-inflammatory diseases and cancer.

Classes IPC  ?

• C07K 19/00 - Peptides hybrides
• A61K 9/14 - Préparations médicinales caractérisées par un aspect particulier à l'état particulaire, p. ex. poudres
• A61K 9/50 - Microcapsules
• C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
• C07K 14/715 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des cytokinesRécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des lymphokinesRécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire pour des interférons
• C12N 15/62 - Séquences d'ADN codant pour des protéines de fusion
• C12P 21/00 - Préparation de peptides ou de protéines