The present invention relates to small interfering RNA (siRNA) molecules against the HTT gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating Huntington's Disease (HD) using the siRNA molecules and AAV vectors.
A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 25/14 - Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/63 - Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
2.
AADC POLYNUCLEOTIDES FOR THE TREATMENT OF PARKINSON'S DISEASE
The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment of Parkinson's Disease.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 25/14 - Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
C12N 7/00 - Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
The present disclosure provides anti-tau antibodies and vectorization thereof (e.g., into AAV particles). Also provided are methods of using anti-tau antibodies and/or AAV particles for prevention, treatment, and/or diagnosis of neurological indications.
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
A61K 39/00 - Medicinal preparations containing antigens or antibodies
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61K 9/00 - Medicinal preparations characterised by special physical form
C07K 7/06 - Linear peptides containing only normal peptide links having 5 to 11 amino acids
C07K 14/005 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
The disclosure relates to small interfering RNA (siRNA) molecules targeting MAPT and adeno-associated viral (AAV) particles encoding the same for treating tauopathies.
C07K 14/47 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from humans from vertebrates from mammals
e.g.e.g., reducing or eliminating, the expression of mutated DMPK via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having myotonic dystrophy type 1 or another DMPK-related disorder.
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to human tau.
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
C07K 14/005 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
C40B 50/04 - Methods of creating libraries, e.g. combinatorial synthesis using dynamic combinatorial chemistry techniques
9.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISORDERS RELATED TO FRATAXIN DEFICIENCY
inter aliae.g.inter aliainter alia, in the treatment of subjects who have, have been diagnosed with, or suspected of having a disorder associated with frataxin (FXN) deficiency, e.g., Friedreich's Ataxia.
inter aliae.g.inter aliainter alia, in the treatment of subjects who have, have been diagnosed with, or are at risk of having a GBA1-related disorder, e.g., Parkinson's Disease (PD), Gaucher Disease (GD), Parkinson's Disease Dementia (PDD), Dementia with Lewy Bodies (DLB), or Lewy Body Dementia (LBD).
e.g.e.g., enhancing, the expression of CDKL5 proteins via delivery using an adeno-associated virus (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having CDD, or another CDKL5-related disorder.
e.g.e.g., enhancing, the expression of STXBP1 proteins via delivery using an adeno-associated virus (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having STXBP1 encephalopathy, or another STXBP1-related disorder.
e.g.e.g., reducing or eliminating, the expression of ATXN2 via delivery of a modulatory polynucleotide using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having, spinocerebellar ataxia type 2 or another ATXN2-related disorder.
The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/005 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
C12N 7/00 - Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.
A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
A61K 9/00 - Medicinal preparations characterised by special physical form
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/63 - Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
45 - Legal and security services; personal services for individuals.
Goods & Services
Biological preparations for gene therapy; adeno-associated
virus (AAY) gene therapy preparations and pharmaceuticals;
biological preparations for treatment of neurological
diseases and disorders; antibody and gene therapy
preparations for treatment of disease, namely, neurological
diseases; medical preparations and pharmaceuticals for
treatment of neurological diseases via antibody, gene
replacement, gene knockdown, or vectorized antibodies;
antibody and gene therapy preparations for neurological
diseases; nucleic acids encoding polypeptides for gene
therapy; drug delivery agents comprising compounds that
facilitate delivery of a wide range of pharmaceuticals and
biotherapeutics; drug delivery agents comprising capsids
viral platforms, and non-viral platforms, that facilitate
delivery of a wide range of pharmaceuticals and
biotherapeutics; pharmaceutical for the treatment of
neurodegenerative diseases and diseases of the central
nervous system, alzheimer's disease, amyotrophic lateral
sclerosis (als), parkinson's disease, friedreich's ataxia,
huntington's disease, and prion disease. Biotechnology research services; research and development of
platform technologies for delivery of therapies; research
and development of platform technologies for delivery of
gene therapies, antibody therapies and pharmaceuticals;
research and development regarding the use of rna-based
functional screening platforms to discover, research and
develop novel capsids for genetic delivery of therapies and
pharmaceuticals; pharmaceutical research and development in
the field of gene therapy and antibody therapy;
pharmaceutical research and development for the treatment of
neurological diseases and disorders; scientific research in
the field of gene therapy and antibody therapy;
pharmaceutical research services; research and development
of pharmaceutical preparations for non-viral and gene
therapy; research and development of pharmaceutical
preparations for neurological diseases; biotechnology
research services; research and development in the
pharmaceutical and biotechnology fields; research and
development regarding the delivery of therapies across the
blood-brain barrier; research and development regarding the
non-viral and viral vectors for the delivery of therapeutic
agents; research and development regarding the
identification and targeting of specific cells and tissues
for gene therapy; providing medical and scientific research
information in the field of clinical trials and gene
therapy; providing a website featuring medical information
and medical research; providing scientific research
information in the field of clinical trials; hosting a
website featuring medical research information. Medical services provided for clinical trials; providing
patient care for clinical trials; providing medical
information in the field of gene therapy; medical treatment
of neurological diseases, neurodegenerative diseases, and
diseases of the central nervous system, including
alzheimer's disease, amyotrophic lateral sclerosis (als),
parkinson's disease, friedreich's ataxia, and huntington's
disease, and prion disease. Collaboration services in the field of platform technologies
for delivery of therapies and pharmaceuticals relating to
intellectual property, namely, licensing of intellectual
property.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Biological preparations for gene therapy; adeno-associated
virus (aav) gene therapy preparations and pharmaceuticals;
biological preparations for treatment of neurological
diseases and disorders; gene therapy preparations for
treatment of disease, namely, neurological diseases; medical
preparations and pharmaceuticals for treatment of
neurological diseases via gene replacement, gene knockdown,
or vectorized antibodies; gene therapy preparations for
neurological diseases; nucleic acids encoding polypeptides
for gene therapy; drug delivery agents comprising compounds
that facilitate delivery of a wide range of pharmaceuticals
and biotherapeutics; drug delivery agents comprising capsids
that facilitate delivery of a wide range of pharmaceuticals
and biotherapeutics. Pharmaceutical research and development in the field of gene
therapy; pharmaceutical research and development for the
treatment of neurological diseases and disorders; scientific
research in the field of gene therapy; pharmaceutical
research services; research and development of
pharmaceutical preparations for gene therapy; research and
development of pharmaceutical preparations for neurological
diseases; biotechnology research services; research and
development of platform technologies for genetic delivery of
therapies and pharmaceuticals; research and development
services in the field of platforms technologies for genetic
delivery of therapies and pharmaceuticals; research and
development in the pharmaceutical and biotechnology fields;
research and development regarding the use of rna-derived
functional screening platforms to discover, research and
develop novel capsids for genetic deliver of therapies and
pharmaceuticals; research and development regarding the
identification and targeting of specific cells and tissues
for gene therapy delivery. Medical treatment of neurological diseases via gene
replacement, gene knockdown, or vectorized antibodies,
namely, Parkinson's disease, Huntington's disease,
Friedreich's ataxia, amyotrophic lateral sclerosis (ALS),
Alzheimer's disease, tauopathies, synucleinopathies, and
neurodegenerative disease.
The present disclosure relates to compositions, methods and processes for the formulation and for the administration of a gene therapy agent using parvovirus e.g., adeno-associated virus (AAV) to the CNS, CNS tissues, CNS structures or CNS cells.
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/47 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from humans from vertebrates from mammals
20.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS RELATED TO GLUCOSYLCERAMIDASE BETA DEFICIENCY
The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of GCase proteins, whether in vitro and/or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson Disease or related condition resulting from a deficiency in the quantity and/or function of GBA gene product or associated with decreased expression or protein levels of GCase protein.
The disclosure provides variant viral genomes, e.g., variant baculovirus genomes, and expression constructs for the variant genomes, as well as methods for producing the same. The variant viral genomes, e.g., variant baculovirus genomes, can be used, for example, to produce recombinant adeno- associated virus (AAV) particles.
e.g.in vitroin vivoin vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson's Disease (PD), Gaucher Disease (GD), Dementia with Lewy Bodies (DLB), or related condition resulting from a deficiency in the quantity and/or function of GBA1 gene product or associated with decreased expression or protein levels of GCase protein.
C12N 9/24 - Hydrolases (3.) acting on glycosyl compounds (3.2)
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The disclosure relates to small interfering RNA (siRNA) molecules targeting MAPT and adeno-associated viral (AAV) particles encoding the same for treating tauopathies.
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
(1) Biological preparations for gene therapy; adeno-associated virus (aav) gene therapy preparations and pharmaceuticals; biological preparations for treatment of neurological diseases and disorders; gene therapy preparations for treatment of disease, namely, neurological diseases; medical preparations and pharmaceuticals for treatment of neurological diseases via gene replacement, gene knockdown, or vectorized antibodies; gene therapy preparations for neurological diseases; nucleic acids encoding polypeptides for gene therapy; drug delivery agents comprising compounds that facilitate delivery of a wide range of pharmaceuticals and biotherapeutics; drug delivery agents comprising capsids that facilitate delivery of a wide range of pharmaceuticals and biotherapeutics. (1) Pharmaceutical research and development in the field of gene therapy; pharmaceutical research and development for the treatment of neurological diseases and disorders; scientific research in the field of gene therapy; pharmaceutical research services; research and development of pharmaceutical preparations for gene therapy; research and development of pharmaceutical preparations for neurological diseases; biotechnology research services; research and development of platform technologies for genetic delivery of therapies and pharmaceuticals; research and development services in the field of platforms technologies for genetic delivery of therapies and pharmaceuticals; research and development in the pharmaceutical and biotechnology fields; research and development regarding the use of rna-derived functional screening platforms to discover, research and develop novel capsids for genetic deliver of therapies and pharmaceuticals; research and development regarding the identification and targeting of specific cells and tissues for gene therapy delivery.
(2) Medical treatment of neurological diseases via gene replacement, gene knockdown, or vectorized antibodies, namely, Parkinson's disease, Huntington's disease, Friedreich's ataxia, amyotrophic lateral sclerosis (ALS), Alzheimer's disease, tauopathies, synucleinopathies, and neurodegenerative disease.
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, including recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Spodoptera frugiperda insect cells (such as Sf9 or Sf21) as viral production cells (VPCs).
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, including recombinant adeno-associated virus (rAAV) particles. The production process and system use Baculoviral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of AAV particles (e.g., rAAVs) which allow for the controlled expression of AAV structural (e.g., capsid) proteins, such as VP1, VP2, and VPS and the controlled expression of AAV nonstructural (e.g., replication) proteins, such as Rep78 and Rep52.
The disclosure provides compositions and methods for the Vectored Augmentation of the Destruction, Expression and/or Regulation of proteins, e.g., VA-DER systems and methods.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 16/00 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies
The present disclosure provides anti-tau antibodies and vectorization thereof (e.g., into AAV particles). Also provided are methods of using anti-tau antibodies and/or AAV particles for prevention, treatment, and/or diagnosis of neurological indications.
C07K 16/08 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from viruses
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present disclosure provides anti-tau antibodies. Also provided are methods of using anti-tau antibodies for treatment and diagnosis of neurological indications.
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C12N 15/63 - Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
33.
COMPOSITIONS AND METHODS FOR CROSSING THE BLOOD BRAIN BARRIER
The disclosure relates to compositions and methods for the preparation, use, and/or formulation of active agents conjugated to ligands for increased crossing of the blood brain barrier.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
35.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF TAUOPATHY
The disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
C07K 14/005 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
The present disclosure provides anti-tau antibodies and adeno-associated virus (AAV) particles with an AAV genome encoding anti-tau antibodies. Also provided are methods of using anti-tau antibodies and AAV particles for treatment and diagnosis of neurological indications.
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C07K 16/00 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
A61K 39/00 - Medicinal preparations containing antigens or antibodies
38.
COMPOSITIONS AND METHODS OF TREATING HUNTINGTON'S DISEASE
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an SMN protein, e.g., an SMN1 protein.
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to HER2.
C07K 14/005 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
C07K 16/32 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products from oncogenes
42.
Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
The present invention relates to small interfering RNA (siRNA) molecules against the SOD1 gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS) using the siRNA molecules and AAV vectors.
The present invention relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and/or canine degenerative myelopathy (DM).
C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C07H 21/02 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
The invention provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, compositions, and formulations, including recombinant adeno-associated viruses (rAAV). The present disclosure presents cell culture mediums for use in producing adeno-associated viruses (AAV), such as AAV which comprise a polynucleotide encoding a payload. In certain embodiments, the cell culture medium comprises a hydrolysate mixture, L-glutamine, poloxamer 188 (e.g., 10% pluronic F-68), a lipid emulsion, and a cholesterol mixture. In certain embodiments, the production process and system use Spodoptera fmgiperda insect cells (such as Sf9 or Sf21) as viral production cells (VPCs). In certain embodiments, the production process and system use Baculo viral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of rAAVs.
The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS).
The disclosure provides compositions and methods for altering, e.g., enhancing, the expression of NPC1 and/or NPC2 proteins, whether in vitro and/or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having NPC1 disease or related condition resulting from a deficiency in the quantity and/or function of NPC1 and/or NPC2 proteins or associated with decreased expression or protein levels of NPC1 and/or NPC2 proteins.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
45 - Legal and security services; personal services for individuals.
Goods & Services
Biological preparations for gene therapy; adeno-associated virus (AAV) gene therapy preparations and pharmaceuticals; Biological preparations for treatment of neurological diseases and disorders; antibody and gene therapy preparations for treatment of disease, namely, neurological diseases; medical preparations and pharmaceuticals for treatment of neurological diseases via antibody, gene replacement, gene knockdown, or vectorized antibodies; antibody and gene therapy preparations for neurological diseases; nucleic acids encoding polypeptides for gene therapy; Drug delivery agents comprising compounds that facilitate delivery of a wide range of pharmaceuticals and biotherapeutics; Drug delivery agents comprising capsids viral platforms, and non-viral platforms, that facilitate delivery of a wide range of pharmaceuticals and biotherapeutics; pharmaceutical for the treatment of neurodegenerative diseases and diseases of the central nervous system, Alzheimer's disease, amyotrophic lateral sclerosis (ALS), Parkinson's disease, Friedreich's Ataxia, Huntington's disease, and prion disease Biotechnology research services; research and development of platform technologies for delivery of therapies; research and development of platform technologies for delivery of gene therapies, antibody therapies and pharmaceuticals; research and development regarding the use of RNA-based functional screening platforms to discover, research and develop novel capsids for genetic delivery of therapies and pharmaceuticals; Pharmaceutical research and development in the field of gene therapy and antibody therapy; Pharmaceutical research and development for the treatment of neurological diseases and disorders; scientific research in the field of gene therapy and antibody therapy; pharmaceutical research services; research and development of pharmaceutical preparations for non-viral and gene therapy; research and development of pharmaceutical preparations for neurological diseases; biotechnology research services; research and development in the pharmaceutical and biotechnology fields; research and development regarding the delivery of therapies across the blood-brain barrier; research and development regarding the non-viral and viral vectors for the delivery of therapeutic agents; research and development regarding the identification and targeting of specific cells and tissues for gene therapy; providing scientific research information in the field of clinical trials; hosting a website featuring medical research information Medical services provided for clinical trials; providing patient care for clinical trials; providing medical
information in the field of
gene therapy; providing a website featuring medical information;
medical treatment of neurological diseases, neurodegenerative diseases, and diseases of the central nervous system, including Alzheimer's disease, amyotrophic lateral sclerosis (ALS), Parkinson's disease, Friedreich's Ataxia, and Huntington's disease, and prion disease Collaboration services in the field of platform technologies for delivery of therapies and pharmaceuticals, namely, licensing of intellectual property
C07K 14/015 - Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
C12N 15/35 - Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
C07K 7/04 - Linear peptides containing only normal peptide links
C12N 15/863 - Poxviral vectors, e.g. vaccinia virus
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C40B 40/08 - Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
The present disclosure describes methods and systems for use in the production of recombinant adeno-associated virus (rAAV) particles comprising a payload (e.g., a polynucleotide encoding aromatic L-amino acid decarboxylase (AADC) or a functional variant thereof). In certain embodiments, the production process uses Sf9 insect cells as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of rAAV particles.
The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of GCase proteins, whether in vitro and/or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson Disease or related condition resulting from a deficiency in the quantity and/or function of GBA gene product or associated with decreased expression or protein levels of GCase protein.
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts tor enhanced tropism to a target tissue.
The present disclosure provides anti-tau antibodies and vectorization thereof (e.g., into AAV particles). Also provided are methods of using anti-tau antibodies and/or AAV particles for prevention, treatment, and/or diagnosis of neurological indications.
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
A61K 39/395 - Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The disclosure relates to compositions, formulations, and methods for the preparation, use, and/or formulation of adeno-associated virus capsid protein variants.
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.
A61K 31/00 - Medicinal preparations containing organic active ingredients
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/00 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
57.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF TAU-RELATED DISORDERS
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to tau.
A61K 39/00 - Medicinal preparations containing antigens or antibodies
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C07K 16/00 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies
A61P 25/00 - Drugs for disorders of the nervous system
58.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS RELATED TO GLUCOSYLCERAMIDASE BETA DEFICIENCY
e.g.in vitroin vivoin vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson Disease or related condition resulting from a deficiency in the quantity and/or function of GBA gene product or associated with decreased expression or protein levels of GCase protein.
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.
The disclosure provides compositions and methods for the preparation, manufacture and use of an adeno-associated virus (AAV) particle for the vectorized delivery of an antibody molecule that binds to HER2.
C07K 16/32 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products from oncogenes
C12N 7/00 - Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
The present disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 25/00 - Drugs for disorders of the nervous system
C07K 14/47 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from humans from vertebrates from mammals
C12N 7/00 - Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
The invention provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like pol peptides, for the prevention and/or treatment of diseases and/or disorders.
The invention provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, including recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Spodoptera frugiperda insect cells (such as Sf9 or Sf21) as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) in the production of AAV particles. In certain embodiments, the production process and system allow for the controlled expression of AAV capsid proteins, such as VP1, VP2 and VP3.
The disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
A61K 39/00 - Medicinal preparations containing antigens or antibodies
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
C12N 7/00 - Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus (AAV) particle comprising a viral gene and a capsid, wherein the viral genome comprises at least one Vectorized Editing of Nucleic acids to correct Overt Mutations (VENOM) element.
The present invention relates to adeno-associated viral (AAV) particles modulatory polynucleotides encoding at least one siRNA molecules and methods of use thereof.
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, compositions and formulations, including recombinant adeno-associated viruses (rAAV). The present disclosure presents cell culture mediums for use in producing adeno-associated viruses (AAV), such as AAV which comprise a polynucleotide encoding a payload. In certain embodiments, the cell culture medium comprises a hydrolysate mixture, L-glutamine, poloxamer 188 (e.g. 10% pluronic F-68), a lipid emulsion, and a cholesterol mixture. In certain embodiments, the production process and system use Spodoptera frugiperda insect cells (such as Sf9 or Sf21) as viral production cells (VPCs). In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of rAAVs.
The present disclosure describes methods and systems for use in the production of adeno- associated virus (AAV) particles, including recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Spodopterafrugiperda insect cells (such as Sf9 or Sf21) as viral production cells (VPCs).
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, including recombinant adeno-associated virus (rAAV) particles. The production process and system use Baculoviral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of AAV particles (e.g., rAAVs) which allow for the controlled expression of AAV structural (e.g., capsid) proteins, such as VP1, VP2, and VPS and the controlled expression of AAV nonstructural (e.g., replication) proteins, such as Rep78 and Rep52.
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.
The present disclosure relates to pharmaceutical compositions of adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
77.
SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
The present disclosure presents methods for producing baculovirus infected insect cells (BIICs). The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, compositions and formulations, including recombinant adeno-associated viruses (rAAV). In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of rAAVs. In certain embodiments, the present disclosure presents methods and systems for designing, producing, clarifying, purifying, formulating, filtering and processing rAAVs and rAAV formulations. In certain embodiments, the production process and system use Spodoptera frugiperda insect cells (such as Sf9 or Sf21) as viral production cells (VPCs).
The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.
C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
C07H 21/02 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/63 - Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present disclosure provides anti-tau antibodies. Also provided are methods of using anti-tau antibodies for treatment and diagnosis of neurological indications.
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
C07K 14/47 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from humans from vertebrates from mammals
80.
CLOSED-ENDED LINEAR DUPLEX DNA FOR NON-VIRAL GENE TRANSFER
Aspects of the disclosure relate to a nucleic acid comprising a heterologous nucleic acid insert flanked by interrupted self-complementary sequences, wherein one self-complementary sequence is interrupted by a cross-arm sequence forming two opposing, lengthwise-symmetric stem-loops, and wherein the other of the self-complementary sequences is interrupted by a truncated cross-arm sequence. Methods of delivering the nucleic acid to a cell are also provided.
C12N 15/63 - Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
C12N 15/66 - General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
C07K 14/005 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
C12N 15/64 - General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
81.
REGULATABLE EXPRESSION USING ADENO-ASSOCIATED VIRUS (AAV)
The present disclosure provides anti-tau antibodies. Also provided are methods of using anti-tau antibodies for treatment and diagnosis of neurological indications.
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
C07K 14/47 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from humans from vertebrates from mammals
83.
TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND DISORDERS ASSOCIATED WITH THE SPINAL CORD
The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
The disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
The invention provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/005 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
C07K 14/47 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from humans from vertebrates from mammals
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, comprising recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Sf9 insect cells as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and Baculoviral Infected Insect Cells (BIICs) in the production of AAV particles.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
89.
AAV capsids with increased tropism to brain tissue
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.
A01N 63/00 - Biocides, pest repellants or attractants, or plant growth regulators containing microorganisms, viruses, microbial fungi, animals or substances produced by, or obtained from, microorganisms, viruses, microbial fungi or animals, e.g. enzymes or fermen
The present disclosure describes methods and systems for use in the production of adeno- associated virus (AAV) particles, compositions, and formulations, including recombinant adeno-associated viruses (rAAV). The present disclosure presents cell culture mediums for use in producing adeno-associated viruses (AAV), such as AAV which comprise a polynucleotide encoding a payload. In certain embodiments, the cell culture medium comprises a hydrolysate mixture, L-glutamine, poloxamer 188 (e.g., 10% pluronic F-68), a lipid emulsion, and a cholesterol mixture. In certain embodiments, the production process and system use Spodoptera fmgiperda insect cells (such as Sf9 or Sf21) as viral production cells (VPCs). In certain embodiments, the production process and system use Baculo viral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of rAAVs.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C12N 7/00 - Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Biological preparations for gene therapy; adeno-associated virus (AAV) gene therapy; Biological preparations for treatment of neurological diseases and disorders; gene therapy preparations for treatment of disease, namely, treatment of disease, namely, neurological diseases; treatments for neurological diseases via gene replacement, gene knockdown, or vectorized antibodies; gene therapy preparations for neurological diseases; polypeptides for gene therapy; Drug delivery agents consisting of compounds that facilitate delivery of a wide range of pharmaceuticals and biotherapeutics; Drug delivery agents consisting of capsids that facilitate delivery of a wide range of pharmaceuticals and biotherapeutics. Pharmaceutical research and development in the field of gene therapy; Pharmaceutical research and development for the treatment of neurological diseases and disorders; scientific research in the field of gene therapy; pharmaceutical research services; research and development of pharmaceutical preparations for gene therapy; research and development of pharmaceutical preparations for neurological diseases; biotechnology research services; research and development of platform technologies for genetic delivery of therapies and pharmaceuticals; research, development, and collaboration services in the field of platforms technologies for genetic delivery of therapies and pharmaceuticals; research and development in the pharmaceutical and biotechnology fields; research and development regarding the use of RNA-based functional screening platforms to discover, research and develop novel capsids for genetic deliver of therapies and pharmaceuticals; research and development regarding the identification and targeting of specific cells and tissues for gene therapy delivery; providing scientific research information in the field of clinical trials and gene therapy; providing medical research. Medical services provided for clinical trials; Providing patient care for clinical trials; Providing medical information in the field of clinical trials and gene therapy; Providing medical information through a website.
92.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS RELATED TO GLUCOSYLCERAMIDASE BETA DEFICIENCY
e.g in vitroin vivoin vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson Disease or related condition resulting from a deficiency in the quantity and/or function of GBA gene product or associated with decreased expression or protein levels of GCase protein.
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
93.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF NIEMANN-PICK TYPE C1 DISEASE
e.g.in vitroin vivoin vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having NPC1 disease or related condition resulting from a deficiency in the quantity and/or function of NPC1 and/or NPC2 proteins or associated with decreased expression or protein levels of NPC1 and/or NPC2 proteins.
C07K 14/47 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from humans from vertebrates from mammals
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of GCase proteins, whether in vitro and/or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson Disease or related condition resulting from a deficiency in the quantity and/or function of GBA gene product or associated with decreased expression or protein levels of GCase protein.
C12N 15/56 - Hydrolases (3) acting on glycosyl compounds (3.2), e.g. amylase, galactosidase, lysozyme
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of frataxin (FXN), whether in vitro and/or in vivo including, but not limited to, the exploitation of engineered promoters. Such compositions include delivery via administration of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Friedreich's ataxia or another neuromuscular or neurological condition resulting from a deficiency in the quantity and/or function of frataxin or associated with decreased expression or protein levels of frataxin.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/47 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from humans from vertebrates from mammals
96.
EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, including recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) in the production of AAV particles. In certain embodiments, the production process and system use Spodoptera frugiperda insect cells (such as Sf9 or Sf21) as viral production cells. In certain embodiments, the production process and system use BEVs which lacks a portion or an entirety of the v-cath gene or includes a mutationally inactivated version of the v-cath gene.
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.
The present disclosure relates to small interfering RNA (siRNA) molecules against the mutant, variant, and/or wild type SCN3A, SCN9A, SCN10A and/or SCN11A gene, adeno-associated viral (AAV) vectors encoding siRNA molecules and methods for treating neuropathic pain using the siRNA molecules and AAV vectors.
The disclosure provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.
C07K 14/005 - Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
C12N 7/00 - Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61K 9/00 - Medicinal preparations characterised by special physical form
The present disclosure relates to cannula systems for delivery of a therapeutic agent to parenchymal tissue of a target subject. In certain embodiments, the cannula system is for delivery of a therapeutic agent to parenchymal tissue in the spine of a target subject. In certain embodiments, the cannula system includes a cannula positioning guide (CPG) useful in the delivery of a therapeutic agent to a localized target tissue of a subject. The guide may be used in a surgical suite or during a surgical procedure.
