Provided herein are expression constructs, nucleic acid vectors, pharmaceutical compositions, and methods for improved expression of functional BEST1 in subjects having mutations in BEST1 and associated retinal dystrophies. Improved expression constructs include BEST 1-targeted short hairpin RNAs (shRNAs), BEST1 coding sequences, promoters driving expression of shRNAs and coding sequences, and/or additional regulatory elements to improve BEST1 expression. Expression constructs, nucleic acid vectors and pharmaceutical compositions thereof, and methods of use thereof, can provide effective treatments for BEST1-associated disorders, such as autosomal dominant bestrophinopathies.
Disclosed arc vectors including i) a first region of nucleic acid sequence comprising a gene of interest, a telomerase occupancy site, and 5' and 3' ends; and ii) a spacer region that is less than 1000 bp links the 5' and 3' ends of the first region of nucleic acid sequence. Vectors may also include a bacterial replication origin or a nucleic acid sequence encoding a RNA selectable marker. Vectors may be double-tranded, linear, close-ended nucleic acid vector. Also disclosed are methods for producing double-stranded, linear, close-ended nucleic acid vectors.
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
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 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
C12N 15/74 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes procaryotes autres que E. coli, p. ex. Lactobacillus, Micromonospora
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
3.
CELL-FREE METHOD OF PRODUCING SYNTHETIC CIRCULAR NUCLEIC ACID
C12Q 1/25 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir des enzymes qui ne peuvent pas être classées dans les groupes
C12Q 1/48 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir une transférase
C12Q 1/533 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir une isomérase
The present disclosure provides a method of producing cyclic dinucleotides (CDNs) on a commercial scale. Also provided are pharmaceutical compositions comprising a purified CDN preparation and use thereof to stimulate the immune system in a subject.
Provided herein are constructs for improved expression of transgenes (e.g., ocular transgenes), nucleic acid vectors thereof, pharmaceutical compositions thereof, and methods of use thereof (e.g., methods of treatment). The expression constructs include regulatory elements such as promoters, enhancers, insulators, and intronic sequences. Expression constructs, nucleic acid vectors and pharmaceutical compositions thereof, and methods of use thereof, disclosed herein can provide effective, durable treatments for ocular diseases.
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 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
The invention features polypeptides containing nuclear localization sequences that can be used to deliver polynucleotides to a cell. The polypeptides can be formulated with a polynucleotide and a lipid.
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 9/1272 - Liposomes non conventionnels, p. ex. liposomes modifiés par un PEG ou liposomes enduits de ou greffés avec des polymères comprenant des agents tensioactifs non phosphatidyliques comme substances formant des bicouches, p. ex. lipides cationiques ou liposomes non phosphatidyliques enduits de ou greffés avec des polymères
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 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
G01N 33/68 - Analyse chimique de matériau biologique, p. ex. de sang ou d'urineTest par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligandsTest immunologique faisant intervenir des protéines, peptides ou amino-acides
The present disclosure provides methods, kits, and systems to efficiently identify the length of poly(A) tails, including exposing an RNA sample to a cleaving enzyme to generate a mixture of nucleotide monophosphates comprising adenosine monophosphate (AMP), cytidine monophosphate (CMP), uridine monophosphate (UMP), and guanosine monophosphate (GMP). Performing reverse-phase high-performance liquid chromatography (HPLC) on the mixture of nucleotide monophosphates. Determining a length of the poly(A) tail of the RNA sample by comparing a peak area of adenosine monophosphate (AMP) from the RNA sample to a peak area of at least one of a cytidine monophosphate (CMP), a uridine monophosphate (UMP), and a guanosine monophosphate (GMP) from the RNA sample.
A method for improving the replication of a covalently closed circular plasmid is provided. The method includes providing a covalently closed circular plasmid having a Pol I- dependent origin of replication, and an insert including a structured DNA sequence selected from inverted repeat sequence, direct repeat sequence, homopolymeric repeat sequence, eukaryotic origin of replication or eukaryotic promoter enhancer sequence, wherein the structured DNA sequence is located at a distance of less than 1000 bp from the Pol I-dependent origin of replication in the direction of replication. The method also includes modifying the covalently closed circular recombinant molecule such that the Pol I-dependent origin of replication is replaced with a Pol Ill-dependent origin of replication, whereby the resultant Pol Ill-dependent origin of replication covalently closed circular plasmid has improved replication. An antibiotic marker free covalently closed circular recombinant DNA molecule is also provided. Recombinant DNA molecules and methods related thereto which include one or more primosomal sites and/ or a selectable marker oritented to transcribe away from a structured DNA sequence are also provided.
C12Q 1/25 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir des enzymes qui ne peuvent pas être classées dans les groupes
C12Q 1/48 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir une transférase
C12Q 1/533 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions faisant intervenir une isomérase
11.
PRODUCTION OF GENE THERAPY VECTOR IN ENGINEERED BACTERIA
Provided herein are improved methods of engineering bacterial cells for production of circular DNA vectors, circular DNA vectors produced by such methods, and pharmaceutical compositions containing such circular DNA vectors. Methods provided herein are amenable to large scale production of high-purity compositions of circular DNA vectors.
The invention provides devices and systems that help deliver agents of interest, such as therapeutic agents (e.g., nucleic acid vectors) to cells of hollow tissues, such as lung. Also provided herein are methods of using such devices, including methods of treating diseases by delivering therapeutic agents to hollow tissues.
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Reagents for use in genetic research; Reagents for scientific or medical research use; Biochemicals, namely deoxyribonucleic acids for in vitro, in vivo and ex vivo scientific and research use; diagnostic preparations for scientific and research use; diagnostic reagents for scientific and research use; nucleic acids for scientific and research use; biochemicals, namely deoxyribonucleic acids for use in biotechnological product development; all of the aforementioned specifically for use in DNA production. Manufacturing services for others in the field of biologics for research, clinical and diagnostic applications, namely, the manufacture of DNA. Biotechnology laboratory services, namely, custom DNA production.
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Reagents for use in genetic research; Reagents for scientific or medical research use; Biochemicals, namely deoxyribonucleic acids for in vitro, in vivo and ex vivo scientific and research use; diagnostic preparations for scientific and research use; diagnostic reagents for scientific and research use; nucleic acids for scientific and research use; biochemicals, namely deoxyribonucleic acids for use in biotechnological product development; all of the aforementioned specifically for use in DNA production. Manufacturing services for others in the field of biologics for research, clinical and diagnostic applications, namely, the manufacture of DNA. Biotechnology laboratory services, namely, custom DNA production
Provided herein are methods of delivering therapeutic agents, such as synthetic circular DNA vectors, to target airway cells (e.g., airway epithelial cells). Such methods can be useful in treating respiratory diseases or disorders, such as cystic fibrosis.
C12N 15/87 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61N 1/32 - Application de courants électriques par électrodes de contact courants alternatifs ou intermittents
C07K 14/705 - RécepteursAntigènes de surface cellulaireDéterminants de surface cellulaire
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
16.
GENE THERAPY VECTORS AND METHODS OF PRODUCTION AND USE THEREOF
Provided herein are improved DNA vectors (e.g., circular DNA vectors and/or non-viral DNA vectors), which can provide features including enhanced expression, persistence, safety, and manufacturability. Also provided herein are methods of producing such DNA vectors, host cells containing such DNA vectors (e.g., engineered bacterial cells useful in production of such DNA vectors), methods of using such DNA vectors (e.g., methods of expressing a therapeutic sequence in a target cell by administering such DNA vectors and methods of treating disease or disorder by administering such DNA vectors), and pharmaceutical compositions containing such DNA vectors.
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
C12N 15/70 - Vecteurs ou systèmes d'expression spécialement adaptés à E. coli
C12N 15/74 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes procaryotes autres que E. coli, p. ex. Lactobacillus, Micromonospora
C12N 15/79 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes
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 1/21 - BactériesLeurs milieux de culture modifiés par l'introduction de matériel génétique étranger
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
Provided herein are improved methods of producing therapeutic circular DNA vectors, pharmaceutical compositions produced by such methods, and methods of using pharmaceutical compositions. The invention is based, at least in part, on cell-free manufacturing processes involving restriction digest and ligation schemes, such as restriction digest processes involving type IIs restriction enzymes. Methods provided herein are amenable to large scale production of high-purity compositions of therapeutic circular DNA vectors.
The present invention relates to a novel analytical method for quantifying the concentration of uncomplexed ("free") gRNA present in Cas-gRNA ribonucleoprotein complexes manufactured for genome editing.
G01N 27/26 - Recherche ou analyse des matériaux par l'emploi de moyens électriques, électrochimiques ou magnétiques en recherchant des variables électrochimiquesRecherche ou analyse des matériaux par l'emploi de moyens électriques, électrochimiques ou magnétiques en utilisant l'électrolyse ou l'électrophorèse
The present invention involves therapies for ocular diseases or disorders in individuals. Provided herein are methods of delivering nucleic acid vectors to ocular cells in the anterior segment of the eye (e.g., corneal endothelium, trabecular meshwork, etc.) involving methods of administering nucleic acid vectors to the individual and methods of electrotransfer of nucleic acid vectors to express therapeutic transgenes. Also provided are nucleic acid vectors for ocular disease, such as Fuchs' dystrophy.
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
Provided herein are isolated DNA vectors comprising a heterologous gene, wherein the DNA vector is devoid of bacterial plasmid DNA and/or bacterial signatures, which can abrogate persistence in vivo. The invention also features pharmaceutical compositions (non-immunogenic pharmaceutical compositions) including the DNA vectors of the invention, which can be used for induction of long-term, episomal expression of a heterologous gene in a subject. The invention involves methods of treating a subject by administering the DNA vectors of the invention, including methods of treating disorders associated with a defect in a target gene.
A61K 31/713 - Acides nucléiques ou oligonucléotides à structure en double-hélice
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
21.
REPLICATIVE MINICIRCLE VECTORS WITH IMPROVED EXPRESSION
A eukaryotic replicative pUC-free minicircle expression vector is provided. The eukaryotic replicative pUC-free minicircle expression vector includes a pUC-free eukaryotic region sequence encoding a transgene of interest and comprising 5′ and 3′ ends and a ii) pUC-free spacer region of less than 500 basepairs in length linking the 5′ and 3′ ends of the eukaryotic region sequences and comprising a bacterial R6K replication origin having at least 95% sequence identity to SEQ ID NO: 11 and SEQ ID NO: 12 and a RNA selectable marker, the RNA selectable marker being an RNA-IN regulating RNA-OUT functional variant having at least 95% sequence identity to SEQ ID NO: 20 or SEQ ID NO: 22.
C12N 15/79 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes
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 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
C12N 15/67 - Méthodes générales pour favoriser l'expression
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C12P 19/34 - Polynucléotides, p. ex. acides nucléiques, oligoribonucléotides
C12P 21/00 - Préparation de peptides ou de protéines
Provided herein are electrotransfer devices for delivering agents (e.g., nucleic acid vectors) to target cells. Also provided are methods of electrotransfer of therapeutic agents.
A61F 9/00 - Procédés ou dispositifs pour le traitement des yeuxDispositifs pour mettre en place des verres de contactDispositifs pour corriger le strabismeAppareils pour guider les aveuglesDispositifs protecteurs pour les yeux, portés sur le corps ou dans la main
A61N 1/05 - Électrodes à implanter ou à introduire dans le corps, p. ex. électrode cardiaque
A61N 1/32 - Application de courants électriques par électrodes de contact courants alternatifs ou intermittents
The present invention involves nucleic acid vectors (e.g., circular DNA vectors) and compositions thereof. Also provided herein are methods of administering nucleic acid vectors (e.g., circular DNA vectors) and compositions thereof, e.g., in combination with pulsed electric field therapy.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A chromatography column stand includes a floor portion, a wall portion, an internal volume, and a base portion. The floor portion supports a chromatography column. The wall portion extends from a first side of the floor portion around a periphery of the floor portion, and has an internal surface. The internal volume is defined by the internal surface of the wall portion and the first side of the floor portion. The base portion extends from an external surface of the stand. The wall portion and the base portion maintain the chromatography column in an upright position when the chromatography column is disposed within the internal volume.
B01D 15/22 - Adsorption sélective, p. ex. chromatographie caractérisée par des caractéristiques de structure ou de fonctionnement relatives à la structure de la colonne
Provided herein are expression constructs, nucleic acid vectors, pharmaceutical compositions, and methods for improved expression of functional BEST1 in subjects having mutations in BEST1 and associated retinal dystrophies. Improved expression constructs include BEST 1 -targeted short hairpin RNAs (shRNAs), BEST1 coding sequences, promoters driving expression of shRNAs and coding sequences, and/or additional regulatory elements to improve BEST1 expression. Expression constructs, nucleic acid vectors and pharmaceutical compositions thereof, and methods of use thereof, can provide effective treatments for BESTl-associated disorders, such as autosomal dominant bestrophinopathies.
C12N 15/87 - Introduction de matériel génétique étranger utilisant des procédés non prévus ailleurs, p. ex. co-transformation
A61F 9/00 - Procédés ou dispositifs pour le traitement des yeuxDispositifs pour mettre en place des verres de contactDispositifs pour corriger le strabismeAppareils pour guider les aveuglesDispositifs protecteurs pour les yeux, portés sur le corps ou dans la main
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 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
The present disclosure provides a method of producing cyclic dinucleotides (CDNs) on a commercial scale. Also provided are pharmaceutical compositions comprising a purified CDN preparation and use thereof to stimulate the immune system in a subject.
The present disclosure relates to a peptide and its analogs that selectively inhibit the NaV1.7 sodium channel. The present disclosure also relates to pharmaceutical compositions useful for treating or preventing a disorder responsive to the blockade of sodium ion channels, especially NaV1.7 sodium ion channels. The present disclosure provides methods of treating a disorder responsive to the blockade of sodium channels, and particularly NaV1.7 sodium channels, in a mammals. The present disclosure further provides compositions and methods for providing analgesia by administering a peptide of the invention.
C07K 14/435 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
The present disclosure provides novel combinations of muscle-specific enhancers and promoters useful for achieving high and persistent expression in muscle tissue or myocytes. The muscle-specific promoter elements are derived from a desmin promoter. The muscle-specific enhancer elements are derived from a desmin promoter and a muscle creatine kinase enhancer.
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 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C12N 15/67 - Méthodes générales pour favoriser l'expression
Provided herein are isolated DNA vectors comprising a heterologous gene, wherein the DNA vector is devoid of bacterial plasmid DNA and/or bacterial signatures, which can abrogate persistence in vivo. The invention also features pharmaceutical compositions (non-immunogenic pharmaceutical compositions) including the DNA vectors of the invention, which can be used for induction of long-term, episomal expression of a heterologous gene in a subject. The invention involves methods of treating a subject by administering the DNA vectors of the invention, including methods of treating disorders associated with a defect in a target gene.
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 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
The present disclosure provides engineered E. coli host cells that combine a knockout of SbcC, SbcD, or both without certain other mutations that can be used to propogate vectors. Methods of improved vector production using such engineered E. coli host cells are also provided.
Provided herein are isolated DNA vectors comprising a heterologous gene, wherein the DNA vector is devoid of bacterial plasmid DNA and/or bacterial signatures, which can abrogate persistence in vivo. The invention also features pharmaceutical compositions (non-immunogenic pharmaceutical compositions) including the DNA vectors of the invention, which can be used for induction of long-term, episomal expression of a heterologous gene in a subject. The invention involves methods of treating a subject by administering the DNA vectors of the invention, including methods of treating disorders associated with a defect in a target gene.
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 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
The present invention relates to the production and use of covalently closed circular (ccc) recombinant plasmids, and more particularly to vector modifications that improve expression of said DNA molecules in the target organism.
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C12N 15/70 - Vecteurs ou systèmes d'expression spécialement adaptés à E. coli
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Biological substances, materials, and preparations for manufacture and development of gene therapy products, pharmaceuticals, biopharmaceuticals, and vaccines; cloning vectors, expression vectors and reagents for clinical or research use. Pharmaceutical preparations for detecting or treating genetic predispositions for medical purposes.
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
Produits et services
Biological substances, namely, cellular growth media for use in plasmid DNA manufacturing platform for biotherapeutics research and development; biological substances, namely, culture medium for plasmid DNA manufacturing. Custom manufacturing services relating to biological processes for plasmid DNA manufacturing using biological organisms in the manufacturing process.
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
Produits et services
(1) Biological substances, namely, microorganisms engineered for plasmid DNA manufacturing; Biological substances, namely microbiological culture medium for plasmid DNA manufacturing; Biological substances, namely bacterial culture medium for plasmid DNA manufacturing (1) Biomanufacturing for others, namely, manufacturing of plasmid DNA using biological organisms in the manufacturing process
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
(1) Biological substances, materials, and preparations for manufacture and development of gene therapy products, pharmaceuticals, biopharmaceuticals, and vaccines; cloning vectors, expression vectors and reagents for clinical or research use; pharmaceutical preparations for detecting or treating genetic predispositions for medical purposes
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
Produits et services
(1) Enzymes in the nature of nucleases and nuclease panels for scientific and research purposes; enzymes in the nature of nucleases and nuclease panels derived from biotechnological processes for use in scientific and research purposes; nucleases and nuclease panels for use in genetic research, genome editing, and genetic editing; enzymes in the nature of nucleases for scientific and research purposes
Provided herein are isolated DNA vectors comprising a heterologous gene, wherein the DNA vector is devoid of bacterial plasmid DNA and/or bacterial signatures, which can abrogate persistence in vivo. The invention also features pharmaceutical compositions (non-immunogenic pharmaceutical compositions) including the DNA vectors of the invention, which can be used for induction of long-term, episomal expression of a heterologous gene in a subject. The invention involves methods of treating a subject by administering the DNA vectors of the invention, including methods of treating disorders associated with a defect in a target gene.
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 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
Produits et services
DNA-modifying endonucleases and nucleic acid vectors for scientific and research purposes, namely, engineered endonucleases that function in tandem with a guiding ribonucleic acid molecule to perform in vivo and in vitro modification of target DNA sequences.
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
Produits et services
DNA-modifying endonucleases and nucleic acid vectors for scientific and research purposes, namely, engineered endonucleases that function in tandem with a guiding ribonucleic acid molecule to perform in vivo and in vitro modification of target DNA sequences
The present disclosure relates to a peptide and its analogs that selectively inhibit the NaV1.7 sodium channel. The present also relates to pharmaceutical compositions useful for treating or preventing a disorder responsive to the blockade of sodium ion channels, especially NaV1.7sodium ion channels. The present disclosure provides methods of treating a disorder responsive to the blockade of sodium channels, and particularly NaV1.7 sodium channels, in a mammals. The present disclosure further provides compositions and methods for providing analgesia by administering a peptide of the invention.
A61P 29/00 - Agents analgésiques, antipyrétiques ou anti-inflammatoires non centraux, p. ex. agents antirhumatismauxMédicaments anti-inflammatoires non stéroïdiens [AINS]
C07K 14/435 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Biological preparations for use in industry and science, namely, for manufacture and development of gene therapy products, pharmaceuticals, biopharmaceuticals, and vaccines; Biological preparations for use in industry and science, namely, cloning vectors, expression vectors and reagents for clinical or research use; all the foregoing being gene therapy products Mixed biological preparations, namely, cloning vectors, expression vectors and reagents for the prevention and treatment of infectious diseases; Pharmaceutical preparations for detecting or treating genetic predispositions for medical purposes; all the foregoing being gene therapy products
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
Produits et services
Biological preparations for use in industry and science, namely culture medium for plasmid DNA manufacturing; biological preparations for use in industry and science, namely, plasmid DNA Biomanufacturing for others, namely, manufacturing of plasmid DNA using biological organisms in the manufacturing process
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
Produits et services
nucleases and nuclease panels for scientific and research purposes; nucleases and nuclease panels derived from biotechnological processes for use in scientific and research purposes; nucleases and nuclease panels for use in genetic research, genome editing, and genetic editing; nucleases.
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
Produits et services
Enzymes in the nature of nucleases and nuclease panels for scientific and research purposes; Enzymes in the nature of nucleases and nuclease panels derived from biotechnological processes for use in industry; Assays and reagents in the nature of nucleases and nuclease panels for use in genetic research, genome editing for research purposes, and genetic editing for research purposes; Enzymes in the nature of nucleases for scientific and research purposes
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
18 - Cuir et imitations du cuir
25 - Vêtements; chaussures; chapellerie
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Biochemicals, namely, ribonucleic acids, deoxyribonucleic acids, peptides, proteins, and antibodies for in vitro, in vivo and ex vivo scientific and research use; diagnostic preparations for scientific and research use; diagnostic reagents for scientific and research use; nucleic acids for scientific and research use; proteins in raw material form for scientific and medical research; enzymes for scientific and research use; biochemicals, namely, monoclonal and polyclonal antibodies for in vitro, in vivo, and ex vivo scientific and research use; laboratory chemicals, namely, an antibody reagent used for the detection of antigens in cell and tissue analysis for in vitro, in vivo, and ex vivo diagnostic use; biochemicals, namely, ribonucleic acids, deoxyribonucleic acids, peptides, proteins, and antibodies for use in biotechnological product development Biological preparations for use in the treatment of cancer, immune response disorders, genetic conditions, infectious diseases, and vascular diseases; diagnostic preparations for medical and veterinary purposes; diagnostic reagents for medical and veterinary purposes; nucleic acid sequences for medical and veterinary purposes; enzymes for medical and veterinary purposes; protein arrays for medical and veterinary diagnosis purposes; antibodies, namely, monoclonal and polyclonal antibodies for the diagnosis, treatment, and prevention of cancer, immune response disorders, genetic conditions, infectious diseases, and vascular diseases Backpacks Clothing, namely, shirts, jackets, sweatshirts Manufacturing services for others in the field of biologics for research, clinical and diagnostic applications; manufacturing services for others, namely, custom production of nucleic acids, peptides and proteins under clinical phase-appropriate quality systems; manufacturing services for others, namely, custom production of nucleic acids, peptides and proteins for use in biotechnological product development; technical consultancy services, namely, providing technical advice and information to others related to the manufacture of biologics for initiating clinical studies Scientific and technological research and development services in the field of nucleic acid, protein, and enzyme activity; scientific and technological research and development services in the field of biologics; design and development of nucleic acids, proteins, and enzymes for use in pharmaceuticals, biotechnology, and medical and veterinary diagnostics
Provided herein are isolated DNA vectors comprising a heterologous gene, wherein the DNA vector is devoid of bacterial plasmid DNA and/or bacterial signatures, which can abrogate persistence in vivo. The invention also features pharmaceutical compositions (non-immunogenic pharmaceutical compositions) including the DNA vectors of the invention, which can be used for induction of long-term, episomal expression of a heterologous gene in a subject. The invention involves methods of treating a subject by administering the DNA vectors of the invention, including methods of treating disorders associated with a defect in a target gene.
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 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
Provided herein are isolated DNA vectors comprising a heterologous gene, wherein the DNA vector is devoid of bacterial plasmid DNA and/or bacterial signatures, which can abrogate persistence in vivo. The invention also features pharmaceutical compositions (non-immunogenic pharmaceutical compositions) including the DNA vectors of the invention, which can be used for induction of long-term, episomal expression of a heterologous gene in a subject. The invention involves methods of treating a subject by administering the DNA vectors of the invention, including methods of treating disorders associated with a defect in a target gene.
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 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
A eukaryotic replicative pUC-free minicircle expression vector is provided. The eukaryotic replicative pUC-free minicircle expression vector includes a pUC-free eukaryotic region sequence encoding a transgene of interest and comprising 5′ and 3′ ends and a ii) pUC-free spacer region of less than 500 basepairs in length linking the 5′ and 3′ ends of the eukaryotic region sequences and comprising a bacterial R6K replication origin having at least 95% sequence identity to SEQ ID NO: 11 and SEQ ID NO: 12 and a RNA selectable marker, the RNA selectable marker being an RNA-IN regulating RNA-OUT functional variant having at least 95% sequence identity to SEQ ID NO: 20 or SEQ ID NO: 22.
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C12N 15/79 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
C12P 21/00 - Préparation de peptides ou de protéines
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C12P 19/34 - Polynucléotides, p. ex. acides nucléiques, oligoribonucléotides
C12N 15/67 - Méthodes générales pour favoriser l'expression
A61K 31/70 - Hydrates de carboneSucresLeurs dérivés
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
54.
Growth of bacterial host cells in gas permeable low-density polyethylene bags for production of plasmid DNA or recombinant molecules
The present disclosure pertains to methods of growing bacterial host cells in a low-density polyethylene (LDPE) bag to produce plasmid DNA or express recombinant protein. The LDPE bag is filled with media, an antibiotic, and host bacterial cells that have been transformed with plasmid DNA encoding a protein of interest. The LDPE bag is sealable to the external environment and incubated at a growth temperature until a desired concentration of bacteria is achieved. When plasmid DNA is desired, host cells are harvested and plasmid DNA is separated from host cell components. When recombinant protein is desired, expression is induced while host cells are in the LDPE bag, followed by the harvest and separation of the recombinant protein. The LDPE bags are sterile and conducive to bacterial growth equal to or greater than that afforded by conventional shake flasks under similar growth conditions.
A method for improving the replication of a covalently closed circular plasmid is provided. The method includes providing a covalently closed circular plasmid having a Pol I-dependent origin of replication, and an insert including a structured DNA sequence selected from inverted repeat sequences, direct repeat sequences, homopolymeric repeat sequences, eukaryotic origins of replication or eukaryotic promoter enhancer sequences, wherein the structured DNA sequence is located at a distance of less than 1000 bp from the Pol I-dependent origin of replication in the direction of replication. The method also includes modifying the covalently closed circular recombinant molecule such that the Pol I-dependent origin of replication is replaced with a Pol III-dependent origin of replication, whereby the resultant Pol III-dependent origin of replication covalently closed circular plasmid has improved replication. An antibiotic marker free covalently closed circular recombinant DNA molecule is also provided.
The present disclosure pertains to methods of growing bacterial host cells in a low-density polyethylene (LDPE) bag to produce plasmid DNA or express recombinant protein. The LDPE bag is filled with media, an antibiotic, and host bacterial cells that have been transformed with plasmid DNA encoding a protein of interest. The LDPE bag is sealable to the external environment and incubated at a growth temperature until a desired concentration of bacteria is achieved. When plasmid DNA is desired, host cells are harvested and plasmid DNA is separated from host cell components. When recombinant protein is desired, expression is induced while host cells are in the LDPE bag, followed by the harvest and separation of the recombinant protein. The LDPE bags are sterile and conducive to bacterial growth equal to or greater than that afforded by conventional shake flasks under similar growth conditions.
The present disclosure pertains to methods of growing bacterial host cells in a low-density polyethylene (LDPE) bag to produce plasmid DNA or express recombinant protein. The LDPE bag is filled with media, an antibiotic, and host bacterial cells that have been transformed with plasmid DNA encoding a protein of interest. The LDPE bag is sealable to the external environment and incubated at a growth temperature until a desired concentration of bacteria is achieved. When plasmid DNA is desired, host cells are harvested and plasmid DNA is separated from host cell components. When recombinant protein is desired, expression is induced while host cells are in the LDPE bag, followed by the harvest and separation of the recombinant protein. The LDPE bags are sterile and conducive to bacterial growth equal to or greater than that afforded by conventional shake flasks under similar growth conditions.
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
C12N 15/65 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression utilisant des marqueurs
C12N 15/70 - Vecteurs ou systèmes d'expression spécialement adaptés à E. coli
C12N 15/52 - Gènes codant pour des enzymes ou des proenzymes
C12M 1/04 - Appareillage pour l'enzymologie ou la microbiologie avec des moyens d'introduction de gaz
The present invention relates to the production and use of covalently closed circular (ccc) recombinant DNA molecules such as plasmids, cosmids, bacterial artificial chromosomes (BACs), bacteriophages, viral vectors and hybrids thereof, and more particularly to vector modifications that improve expression of said DNA molecules.
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C12N 15/79 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
C12P 21/00 - Préparation de peptides ou de protéines
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C12P 19/34 - Polynucléotides, p. ex. acides nucléiques, oligoribonucléotides
C12N 15/67 - Méthodes générales pour favoriser l'expression
A01K 67/00 - Élevage ou obtention d'animaux, non prévus ailleursNouvelles races ou races modifiées d'animaux
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
59.
Replicative minicircle vectors with improved expression
A eukaryotic replicative pUC-free minicircle expression vector is provided. The eukaryotic replicative pUC-free minicircle expression vector includes a pUC-free eukaryotic region sequence encoding a transgene of interest and comprising 5′ and 3′ ends and ii) a pUC-free spacer region of less than 500 basepairs in length linking the 5′ and 3′ ends of the eukaryotic region sequences and comprising a bacterial R6K replication origin having at least 95% sequence identity to SEQ ID NO: 11 or SEQ ID NO: 12 and a RNA selectable marker, the RNA selectable marker being an RNA-IN regulating RNA-OUT functional variant having at least 95% sequence identity to SEQ ID NO:20 or SEQ ID NO:22.
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C12N 15/79 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
C12P 21/00 - Préparation de peptides ou de protéines
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C12P 19/34 - Polynucléotides, p. ex. acides nucléiques, oligoribonucléotides
C12N 15/67 - Méthodes générales pour favoriser l'expression
A01K 67/00 - Élevage ou obtention d'animaux, non prévus ailleursNouvelles races ou races modifiées d'animaux
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
Produits et services
Manufacturing services for others in the field of biologics for research, clinical and diagnostic applications; manufacturing services for others, namely, custom production of nucleic acids, peptides and proteins under clinical phase-appropriate quality systems; manufacturing services for others, namely, custom production of nucleic acids, peptides and proteins for use in biotechnological product development; technical consultancy services, namely, providing technical advice and information to others related to the manufacture of biologics for initiating clinical studies
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
Produits et services
Chemicals used in industry, science and photography, as well as in agriculture, horticulture and forestry; unprocessed artificial resins, unprocessed plastics; manures; fire extinguishing compositions; tempering and soldering preparations; chemical substances for preserving foodstuffs; tanning substances; adhesives used in industry; biologics; biologics for scientific and research use; diagnostic preparations for scientific and research use; diagnostic reagents for scientific and research use; nucleic acids, proteins, enzymes, and antibodies for scientific and research use; biologics for use in biotechnological development. Pharmaceuticals, medical and veterinary preparations; sanitary preparations for medical purposes; dietetic food and substances adapted for medical or veterinary use, food for babies; dietary supplements for humans and animals; plasters, materials for dressings; material for stopping teeth, dental wax; disinfectants; preparations for destroying vermin; fungicides, herbicides; biologics; biologics for medical and veterinary purposes; diagnostic preparations for medical and veterinary purposes; diagnostic reagents for medical and veterinary purposes; nucleic acids, proteins, enzymes, and antibodies for medical and veterinary purposes. Contract manufacturing in the field of biologics for research, clinical, and diagnostic applications including ready-to-use AAV helper, cytokine, and reporter plasmids.
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
Produits et services
(1) Biologics, namely, ribonucleic acids, deoxyribonucleic acids, peptides, proteins, and antibodies for in vitro, in vivo and ex vivo for scientific and research use; diagnostic preparations for scientific and research use; diagnostic reagents for scientific and research use; nucleic acids, proteins, enzymes, and antibodies for scientific and research use; biologics, namely, ribonucleic acids, deoxyribonucleic acids, peptides, proteins, and antibodies for use in biotechnological development
(2) Biological preparations for use in the treatment of cancer, immune response disorders, genetic disorders, infectious diseases, namely, viral and bacterial infections, and vascular diseases for medical and veterinary purposes; diagnostic preparations for medical and veterinary purposes; diagnostic reagents for medical and veterinary purposes; nucleic acids, proteins, enzymes, and antibodies for medical and veterinary purposes (1) Contract manufacturing in the field of biologics, namely, manufacturing of nucleic acids for research, clinical, and diagnostic applications, namely, ready-to-use adeno-associated virus (AAV) helper, cytokine, and reporter plasmids
The present invention relates to the production and use of covalently closed circular (ccc) recombinant plasmids, and more particularly to vector modifications that improve expression of said DNA molecules in the target organism.
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C12N 15/70 - Vecteurs ou systèmes d'expression spécialement adaptés à E. coli
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et services
Manufacturing services for others in the field of biologics for research, clinical, and diagnostic applications; technical consultancy services, namely, providing technical advice and information to others related to the manufacture of biologics for research, clinical, and diagnostic applications Scientific and technological research and development services in the field of nucleic acid, protein, and enzyme activity; scientific and technological research and development services in the field of biologics; design and development of nucleic acids, proteins, and enzymes for use in pharmaceuticals, biotechnology, and medical and veterinary diagnostics
General methods and strains of bacteria are described that dramatically simplify and streamline plasmid DNA production. In one preferred embodiment, endolysin mediated plasmid extraction combined with flocculation mediated removal of cell debris and host nucleic acids achieves increased yield and purity with simplified downstream purification and reduced waste streams, thus reducing production costs.
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Biochemicals, namely, ribonucleic acids, deoxyribonucleic acids, peptides, proteins, and antibodies for in vitro, in vivo and ex vivo scientific and research use; diagnostic preparations for scientific and research use; diagnostic reagents for scientific and research use; nucleic acids for scientific and research use; proteins in raw material form for scientific and medical research; enzymes for scientific and research use; biochemicals, namely, monoclonal and polyclonal antibodies for in vitro, in vivo, and ex vivo scientific and research use; laboratory chemicals, namely, an antibody reagent used for the detection of antigens in cell and tissue analysis for in vitro, in vivo, and ex vivo diagnostic use; biochemicals, namely, ribonucleic acids, deoxyribonucleic acids, peptides, proteins, and antibodies for use in biotechnological product development Biological preparations for use in the treatment of cancer, immune response disorders, genetic conditions, infectious diseases, and vascular diseases; diagnostic preparations for medical and veterinary purposes; diagnostic reagents for medical and veterinary purposes; nucleic acid sequences for medical and veterinary purposes; enzymes for medical and veterinary purposes; protein arrays for medical and veterinary diagnosis purposes; antibodies, namely, monoclonal and polyclonal antibodies for the diagnosis, treatment, and prevention of cancer, immune response disorders, genetic conditions, infectious diseases, and vascular diseases
01 - Produits chimiques destinés à l'industrie, aux sciences ainsi qu'à l'agriculture
05 - Produits pharmaceutiques, vétérinaires et hygièniques
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
Produits et services
Biochemicals, namely, ribonucleic acids, deoxyribonucleic acids, peptides, proteins, and antibodies for in vitro, in vivo and ex vivo scientific and research use; diagnostic preparations for scientific and research use; diagnostic reagents for scientific and research use; nucleic acids for scientific and research use; proteins in raw material form for scientific and medical research; enzymes for scientific and research use; biochemicals, namely, monoclonal and polyclonal antibodies for in vitro, in vivo, and ex vivo scientific and research use; laboratory chemicals, namely, an antibody reagent used for the detection of antigens in cell and tissue analysis for in vitro, in vivo, and ex vivo diagnostic use; biochemicals, namely, ribonucleic acids, deoxyribonucleic acids, peptides, proteins, and antibodies for use in biotechnological product development Biological preparations for use in the treatment of cancer, immune response disorders, genetic conditions, infectious diseases, and vascular diseases; diagnostic preparations for medical and veterinary purposes; diagnostic reagents for medical and veterinary purposes; nucleic acid sequences for medical and veterinary purposes; enzymes for medical and veterinary purposes; protein arrays for medical and veterinary diagnosis purposes; antibodies, namely, monoclonal and polyclonal antibodies for the diagnosis, treatment, and prevention of cancer, immune response disorders, genetic conditions, infectious diseases, and vascular diseases Manufacturing services for others in the field of biologics for research, clinical and diagnostic applications; technical consultancy services, namely, providing technical advice and information to others related to the manufacture of biologics for research, clinical and diagnostic applications
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
Produits et services
Manufacturing services for others in the field of biologics for research, clinical and diagnostic applications; technical consultancy services, namely, providing technical advice and information to others related to the manufacture of biologics for research, clinical and diagnostic applications
69.
Replicative minicircle vectors with improved expression
The present invention relates to the production and use of covalently closed circular (ccc) recombinant DNA molecules such as plasmids, cosmids, bacterial artificial chromosomes (BACs), bacteriophages, viral vectors and hybrids thereof, and more particularly to vector modifications that improve expression of said DNA molecules.
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
C12N 15/79 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
C12P 21/00 - Préparation de peptides ou de protéines
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C12P 19/34 - Polynucléotides, p. ex. acides nucléiques, oligoribonucléotides
C12N 15/67 - Méthodes générales pour favoriser l'expression
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
Improved DNA vaccine plasmids are disclosed that contain novel immunostimulatory RNA compositions. The improved plasmids eliminate all extraneous sequences, incorporate a novel antibiotic free short RNA based selectable marker, increase eukaryotic expression using a novel chimeric promoter, improve yield and stability during bacterial production, and improve immunostimulation. These vectors are utilized in immunization to elicit improved immune responses or therapy to induce type 1 interferon production.
A61K 39/145 - Orthomyxoviridae, p. ex. virus de l'influenza
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
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
C12N 15/117 - Acides nucléiques présentant des propriétés immunomodulatrices, p. ex. contenant des motifs CpG
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
Improved DNA vaccine plasmids are disclosed that contain novel immunostimulatory RNA compositions. The improved plasmids eliminate all extraneous sequences, incorporate a novel antibiotic free short RNA based selectable marker, increase eukaryotic expression using a novel chimeric promoter, improve yield and stability during bacterial production, and improve immunostimulation. These vectors are utilized in immunization to elicit improved immune responses or therapy to induce type 1 interferon production.
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
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 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
C12N 15/70 - Vecteurs ou systèmes d'expression spécialement adaptés à E. coli
C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
The present invention relates to the production and use of covalently closed circular (ccc) recombinant DNA molecules such as plasmids, cosmids, bacterial artificial chromosomes (BACs), bacteriophages, viral vectors and hybrids thereof, and more particularly to vector modifications that improve expression of these DNA molecules.
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
A61K 31/70 - Hydrates de carboneSucresLeurs dérivés
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C12N 15/79 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes
C12N 15/67 - Méthodes générales pour favoriser l'expression
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
A01K 67/00 - Élevage ou obtention d'animaux, non prévus ailleursNouvelles races ou races modifiées d'animaux
General methods and strains of bacteria are described that dramatically simplify and streamline plasmid DNA production. In one preferred embodiment, endolysin mediated plasmid extraction combined with flocculation mediated removal of cell debris and host nucleic acids achieves increased yield and purity with simplified downstream purification and reduced waste streams, thus reducing production costs.
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
C12P 21/04 - Peptides ou polypeptides cycliques ou pontés, p. ex. bacitracine
C12N 15/74 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes procaryotes autres que E. coli, p. ex. Lactobacillus, Micromonospora
General methods and strains of bacteria are described that dramatically simplify and streamline plasmid DNA production. In one preferred embodiment, endolysin mediated plasmid extraction combined with flocculation mediated removal of cell debris and host nucleic acids achieves increased yield and purity with simplified downstream purification and reduced waste streams, thus reducing production costs.
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
C12P 21/04 - Peptides ou polypeptides cycliques ou pontés, p. ex. bacitracine
C12N 15/74 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes procaryotes autres que E. coli, p. ex. Lactobacillus, Micromonospora
The present invention relates to the production and use of covalently closed circular (ccc) recombinant plasmids, and more particularly to vector modifications that improve expression of said DNA molecules in the target organism.
C12N 15/70 - Vecteurs ou systèmes d'expression spécialement adaptés à E. coli
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
76.
Eukaryotic expression vectors resistant to transgene silencing
The present invention relates to recombinant DNA molecules such as plasmids, non-viral vectors, viral vectors and hybrids thereof, and more particularly to vector modifications that improve expression of said DNA molecules in cell lines and organisms.
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
40 - Traitement de matériaux; recyclage, purification de l'air et traitement de l'eau
Produits et services
Contract manufacturing in the field of biologics for research, clinical, and diagnostic applications including ready-to-use AAV helper, cytokine, and reporter plasmids
Improved DNA vaccine plasmids are disclosed that contain novel immunostimulatory RNA compositions. The improved plasmids eliminate all extraneous sequences, incorporate a novel antibiotic free short RNA based selectable marker, increase eukaryotic expression using a novel chimeric promoter, improve yield and stability during bacterial production, and improve immunostimulation. These vectors are utilized in immunization to elicit improved immune responses or therapy to induce type 1 interferon production.
C12N 15/70 - Vecteurs ou systèmes d'expression spécialement adaptés à E. coli
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
The present invention relates to the propagation of covalently closed circular recombinant DNA molecules such as plasmids, cosmids, bacterial artificial chromosomes (BACs), bacteriophages, viral vectors and hybrids thereof, and more particularly is strain modifications that improve strain viability, plasmid stability, plasmid production yield, and plasmid-directed protein production yield, using said DNA molecules in fermentation culture.
The present invention relates to the production of covalently closed circular (ccc) recombinant DNA molecules such as plasmids, cosmids, bacterial artificial chromosomes (BACs), bacteriophages, viral vectors and hybrids thereof, and more particularly to vector modifications that improve production yield of said DNA molecules in fermentation culture.
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
C12N 15/69 - Augmentation du nombre de copies du vecteur
C12N 15/70 - Vecteurs ou systèmes d'expression spécialement adaptés à E. coli
General methods and strains of bacteria are described, that dramatically simplify and streamline plasmid DNA production. In one preferred embodiment, endolysin mediated plasmid extraction combined with flocculation mediated removal of cell debris and host nucleic acids achieves increased yield and purity with simplified downstream purification and reduced waste streams, thus reducing production costs.
Improvements in plasmid DNA production technology are needed to insure the economic feasibility of future DNA vaccines and DNA therapeutics. General methods are described, by means of which it is possible to dramatically increase plasmid DNA productivity. These processes feature RNA based inducers of plasmid copy number.
C12P 19/34 - Polynucléotides, p. ex. acides nucléiques, oligoribonucléotides
C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN
C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
C12N 15/69 - Augmentation du nombre de copies du vecteur
C12N 15/70 - Vecteurs ou systèmes d'expression spécialement adaptés à E. coli
Improved DNA vaccine plasmids are disclosed. The improved plasmids eliminate all extraneous sequences, and have superior eukaryotic expression, improved yield and stability during bacterial production, facilitate flexible targeting of antigens to various intracellular destinations, and novel RNA-based functionality. These vectors are utilized in novel immunization methods wherein combinations of immunostimulatory DNA vaccine plasmids that target antigens to various intracellular destinations are used to elicit improved immune response.
Improvements in plasmid DNA production technology are needed to insure the economic feasibility of future DNA vaccines and DNA therapeutics. General methods are described, by means of which it is possible to dramatically increase plasmid DNA productivity in a fermentor. These processes feature Fed-batch fermentation strategies, combined with novel growth and induction phase temperature shifts.
A general method and strains of bacteria are described, by means of which it is possible to dramatically purify plasmid DNA with respect to genomic DNA, and RNA. In one preferred embodiment, lysis and nuclease removal of host nucleic acids is an integral component of the fermentation/harvest process, and as such, achieves increased yield and purity with simplified downstream purification and reduced waste streams, thus reducing production costs.
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci