LT4005603T - Adenoasocijuoti virionai su variantine kapside ir jų panaudojimo būdai - Google Patents
Adenoasocijuoti virionai su variantine kapside ir jų panaudojimo būdaiInfo
- Publication number
- LT4005603T LT4005603T LTEP21201945.9T LT21201945T LT4005603T LT 4005603 T LT4005603 T LT 4005603T LT 21201945 T LT21201945 T LT 21201945T LT 4005603 T LT4005603 T LT 4005603T
- Authority
- LT
- Lithuania
- Prior art keywords
- adeno
- methods
- associated virus
- variant capsid
- virus virions
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/861—Adenoviral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
- A61P27/06—Antiglaucoma agents or miotics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/70—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving virus or bacteriophage
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/70—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving virus or bacteriophage
- C12Q1/701—Specific hybridization probes
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
- G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
- G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
- G01N33/5044—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
- G01N33/5058—Neurological cells
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/16—Aptamers
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14021—Viruses as such, e.g. new isolates, mutants or their genomic sequences
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/40—Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/158—Expression markers
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/16—Primer sets for multiplex assays
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Immunology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Ophthalmology & Optometry (AREA)
- Gastroenterology & Hepatology (AREA)
- Analytical Chemistry (AREA)
- Urology & Nephrology (AREA)
- Hematology (AREA)
- Cell Biology (AREA)
- Dermatology (AREA)
- Marine Sciences & Fisheries (AREA)
- Diabetes (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161478355P | 2011-04-22 | 2011-04-22 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| LT4005603T true LT4005603T (lt) | 2025-01-10 |
Family
ID=47042176
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| LTEP17168534.0T LT3254703T (lt) | 2011-04-22 | 2012-04-20 | Adenoasocijuoto viruso virionai su variantine kapside ir jų panaudojimo būdai |
| LTEP12774323.5T LT2699270T (lt) | 2011-04-22 | 2012-04-20 | Adeno-asocijuoto viruso virionai su variantine kapside ir jų naudojimo būdai |
| LTEP20151479.1T LT3693025T (lt) | 2011-04-22 | 2012-04-20 | Adeno-asocijuoto viruso virionai su variantine kapside ir jų panaudojimo būdai |
| LTEP21201945.9T LT4005603T (lt) | 2011-04-22 | 2012-04-20 | Adenoasocijuoti virionai su variantine kapside ir jų panaudojimo būdai |
Family Applications Before (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| LTEP17168534.0T LT3254703T (lt) | 2011-04-22 | 2012-04-20 | Adenoasocijuoto viruso virionai su variantine kapside ir jų panaudojimo būdai |
| LTEP12774323.5T LT2699270T (lt) | 2011-04-22 | 2012-04-20 | Adeno-asocijuoto viruso virionai su variantine kapside ir jų naudojimo būdai |
| LTEP20151479.1T LT3693025T (lt) | 2011-04-22 | 2012-04-20 | Adeno-asocijuoto viruso virionai su variantine kapside ir jų panaudojimo būdai |
Country Status (26)
| Country | Link |
|---|---|
| US (9) | US9193956B2 (enExample) |
| EP (5) | EP3254703B1 (enExample) |
| JP (6) | JP6072772B2 (enExample) |
| KR (4) | KR102202908B1 (enExample) |
| CN (3) | CN103561774B (enExample) |
| AU (4) | AU2012245328B2 (enExample) |
| BR (1) | BR112013027120B1 (enExample) |
| CA (2) | CA2833870C (enExample) |
| CY (3) | CY1119765T1 (enExample) |
| DK (4) | DK4005603T3 (enExample) |
| ES (4) | ES2638342T3 (enExample) |
| FI (1) | FI4005603T3 (enExample) |
| HR (4) | HRP20241577T1 (enExample) |
| HU (4) | HUE057102T2 (enExample) |
| IL (2) | IL280819B2 (enExample) |
| LT (4) | LT3254703T (enExample) |
| MX (1) | MX349138B (enExample) |
| PL (4) | PL4005603T3 (enExample) |
| PT (4) | PT4005603T (enExample) |
| RS (4) | RS66184B1 (enExample) |
| RU (2) | RU2611202C2 (enExample) |
| SG (3) | SG10201800873WA (enExample) |
| SI (4) | SI4005603T1 (enExample) |
| SM (4) | SMT202400520T1 (enExample) |
| WO (1) | WO2012145601A2 (enExample) |
| ZA (2) | ZA201307968B (enExample) |
Families Citing this family (293)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| JP6072772B2 (ja) | 2011-04-22 | 2017-02-01 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法 |
| TWI702955B (zh) | 2012-05-15 | 2020-09-01 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| WO2014093709A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| IL239317B (en) | 2012-12-12 | 2022-07-01 | Broad Inst Inc | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| US10266845B2 (en) | 2013-02-08 | 2019-04-23 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| DK2954051T3 (da) | 2013-02-08 | 2019-07-08 | Univ Pennsylvania | Modificeret kapsid til genoverførsel til behandling af nethinden |
| JP6591956B2 (ja) | 2013-03-15 | 2019-10-16 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Mps1を治療するための組成物および方法 |
| AU2014265799B2 (en) | 2013-05-16 | 2019-10-31 | Applied Genetic Technologies Corporation | Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases |
| WO2014194132A1 (en) | 2013-05-31 | 2014-12-04 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| EP3011033B1 (en) | 2013-06-17 | 2020-02-19 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| AU2014281028B2 (en) | 2013-06-17 | 2020-09-10 | Massachusetts Institute Of Technology | Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy |
| WO2014204725A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
| CN105793425B (zh) | 2013-06-17 | 2021-10-26 | 布罗德研究所有限公司 | 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用 |
| CN106062197A (zh) | 2013-06-17 | 2016-10-26 | 布罗德研究所有限公司 | 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化 |
| ES2716615T3 (es) | 2013-06-28 | 2019-06-13 | Inst Nat Sante Rech Med | Métodos para expresar un polinucleótido de interés en la retina de un sujeto |
| ES2794499T5 (en) | 2013-07-08 | 2025-08-12 | Inst Nat Sante Rech Med | Methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereof |
| WO2015038958A1 (en) | 2013-09-13 | 2015-03-19 | California Institute Of Technology | Selective recovery |
| CN118813621A (zh) * | 2013-12-12 | 2024-10-22 | 布罗德研究所有限公司 | 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用 |
| EP3080259B1 (en) | 2013-12-12 | 2023-02-01 | The Broad Institute, Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
| EP3080257A1 (en) | 2013-12-12 | 2016-10-19 | The Broad Institute Inc. | Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders |
| WO2015089486A2 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
| MX374530B (es) | 2013-12-12 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapeuticas de los sistemas y composiciones crispr-cas para actuar sobre hbv y trastornos y enfermedades virales. |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| US20170007720A1 (en) * | 2014-02-21 | 2017-01-12 | University Of Florida Research Foundation, Inc. | Methods and compositions for gene delivery to on bipolar cells |
| EP3113787B1 (en) * | 2014-03-04 | 2019-12-04 | University of Florida Research Foundation, Inc. | Improved raav vectors and methods for transduction of photoreceptors and rpe cells |
| EP3114227B1 (en) | 2014-03-05 | 2021-07-21 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| US9938521B2 (en) | 2014-03-10 | 2018-04-10 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10) |
| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| CA2942324C (en) * | 2014-03-11 | 2024-05-14 | Wayne State University | A modified mglur6 promoter and methods of use |
| AU2015231439B2 (en) | 2014-03-17 | 2019-11-14 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| US11242525B2 (en) | 2014-03-26 | 2022-02-08 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating sickle cell disease |
| WO2015153780A1 (en) | 2014-04-02 | 2015-10-08 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| IL248102B (en) | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN106794262A (zh) * | 2014-07-31 | 2017-05-31 | 俄克拉何马大学董事会 | 哺乳动物rpe65的高异构水解酶活性突变体 |
| US10821193B2 (en) | 2014-09-16 | 2020-11-03 | Genzyme Corporation | Adeno-associated viral vectors for treating myocilin (MYOC) glaucoma |
| RU2020109343A (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| CN112375760A (zh) | 2014-11-14 | 2021-02-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| EP3889260A1 (en) | 2014-12-12 | 2021-10-06 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
| AU2015369725A1 (en) | 2014-12-24 | 2017-06-29 | Massachusetts Institute Of Technology | CRISPR having or associated with destabilization domains |
| WO2016131009A1 (en) * | 2015-02-13 | 2016-08-18 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| US10648000B2 (en) | 2015-02-16 | 2020-05-12 | University Of Florida Research Foundation, Incorporated | rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes |
| WO2016134375A1 (en) * | 2015-02-20 | 2016-08-25 | University Of Iowa Research Foundation | Methods and compositions for treating genetic eye diseases |
| MY187898A (en) * | 2015-03-02 | 2021-10-27 | Adverum Biotechnologies Inc | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| EP3277819B1 (en) | 2015-03-24 | 2021-03-03 | The Regents of The University of California | Adeno-associated virus variants and methods of use thereof |
| US10081659B2 (en) | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| CA2982966C (en) | 2015-04-24 | 2024-02-20 | Editas Medicine, Inc. | Evaluation of cas9 molecule/guide rna molecule complexes |
| EP3294332A4 (en) * | 2015-05-14 | 2018-12-26 | Joslin Diabetes Center, Inc. | Retinol-binding protein 3 (rbp3) as a protective factor in non-diabetic retinal degeneration |
| WO2016205759A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
| WO2016205613A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Crispr enzyme mutations reducing off-target effects |
| CA3004807C (en) * | 2015-12-04 | 2022-02-22 | Universite Pierre Et Marie Curie (Paris 6) | Promoters and uses thereof |
| ES2869284T3 (es) * | 2015-12-11 | 2021-10-25 | California Inst Of Techn | Péptidos de diana para dirigir virus adenoasociados (AAV) |
| EP3387434A1 (en) | 2015-12-11 | 2018-10-17 | The U.S.A. As Represented By The Secretary, Department Of Health And Human Services | Gene therapy for combined methylmalonic acidemia/aciduria and hyperhomocysteinemia/homocystinuria, cobalamin c type, and deficiency of mmachc |
| GB2545763A (en) | 2015-12-23 | 2017-06-28 | Adverum Biotechnologies Inc | Mutant viral capsid libraries and related systems and methods |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
| EP3433364A1 (en) | 2016-03-25 | 2019-01-30 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| IL322403A (en) | 2016-04-15 | 2025-09-01 | Univ Pennsylvania | Preparations for the treatment of wet age-related macular degeneration |
| AU2017256910B2 (en) | 2016-04-29 | 2022-04-07 | Centre National De La Recherche Scientifique | Optogenetic visual restoration using Chrimson |
| WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| ES2908467T3 (es) | 2016-04-29 | 2022-04-29 | Adverum Biotechnologies Inc | Evasión de anticuerpos neutralizantes por un virus adenoasociado recombinante |
| WO2017192750A1 (en) * | 2016-05-04 | 2017-11-09 | Oregon Health & Science University | Recombinant adeno-associated viral vectors |
| US11364308B2 (en) | 2016-05-13 | 2022-06-21 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and methods of use thereof |
| IL302748B1 (en) | 2016-05-18 | 2025-09-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| MA44546B1 (fr) * | 2016-06-15 | 2021-03-31 | Univ California | Virus adéno-associés variants et procédés d'utilisation |
| ES2916344T3 (es) * | 2016-06-16 | 2022-06-30 | Adverum Biotechnologies Inc | Composiciones y métodos para reducir la neovascularización ocular |
| AU2017286673B2 (en) * | 2016-06-16 | 2021-06-24 | Adverum Biotechnologies, Inc. | Treatment of AMD using AAV2 variant with aflibercept |
| MX2019001276A (es) | 2016-07-29 | 2019-06-13 | Univ California | Viriones de virus adenoasociados con cápside variante y métodos para su uso. |
| BR112019001887A2 (pt) | 2016-08-02 | 2019-07-09 | Editas Medicine Inc | composições e métodos para o tratamento de doença associada a cep290 |
| RU2019109021A (ru) | 2016-08-29 | 2020-09-29 | Уэйн Стейт Юниверсити | Идентификация мутаций в вариантах каналопсина, имеющих улучшенную чувствительность к свету, и способы их применения |
| JP2019531787A (ja) | 2016-08-30 | 2019-11-07 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| AU2017372351B2 (en) * | 2016-11-06 | 2019-12-12 | Nanoscope Technologies Llc | Optogenetic modulation by multi-characteristic opsins for vision restoration and other applications thereof |
| US11236360B2 (en) | 2016-12-09 | 2022-02-01 | Regents Of The University Of Minnesota | Adeno-associated viruses engineered for selectable tropism |
| JP2020510424A (ja) * | 2017-02-28 | 2020-04-09 | アドヴェラム バイオテクノロジーズ, インコーポレイテッド | 改変aavカプシドおよびその使用 |
| WO2018170184A1 (en) | 2017-03-14 | 2018-09-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| MX2019011040A (es) | 2017-03-17 | 2020-01-20 | Adverum Biotechnologies Inc | Composiciones y metodos para potenciar la expresion genica. |
| US20200370039A1 (en) * | 2017-04-11 | 2020-11-26 | Ruprecht-Karls-Universitat Heidelberg | Adeno-associated virus library |
| TW201903146A (zh) | 2017-04-14 | 2019-01-16 | 美商銳進科斯生物股份有限公司 | 使用由人類神經或膠細胞產生的重組人類艾杜糖醛酸鹽 (iduronate)-2-硫酸酯酶 (ids) 之黏多醣病ii之治療 |
| US11752181B2 (en) | 2017-05-05 | 2023-09-12 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| WO2018209158A2 (en) | 2017-05-10 | 2018-11-15 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| US20200121746A1 (en) * | 2017-06-30 | 2020-04-23 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsids and methods of use thereof |
| WO2019010335A1 (en) | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I |
| EP3654860A1 (en) | 2017-07-17 | 2020-05-27 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| CA3059995A1 (en) | 2017-08-28 | 2019-03-07 | The Regents Of The University Of California | Adeno-associated virus capsid variants and methods of use thereof |
| DK3684423T5 (da) * | 2017-09-20 | 2024-08-26 | 4D Molecular Therapeutics Inc | Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf |
| WO2019067840A1 (en) | 2017-09-29 | 2019-04-04 | Voyager Therapeutics, Inc. | RESTRICTING CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE FROM FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| KR102776626B1 (ko) * | 2017-10-16 | 2025-03-04 | 비게네론 게엠베하 | Aav 벡터 |
| CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| EP3697449A1 (en) | 2017-10-18 | 2020-08-26 | REGENXBIO Inc. | Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified vegf-trap |
| BR112020007405A2 (pt) | 2017-10-18 | 2020-12-08 | Regenxbio Inc. | Produtos terapêuticos de anticorpo pós-translacionalmente modificados totalmente humanos |
| KR102763312B1 (ko) * | 2017-10-20 | 2025-02-07 | 인쎄름 (엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔) | 치료적으로 유효한 양의 재조합 aav9- 유도 벡터의 망막하 전달을 포함하는 대상체의 원추 광수용체 내 관심의 폴리뉴클레오티드를 발현하는 방법 |
| US11851671B2 (en) | 2017-10-23 | 2023-12-26 | Regents Of The University Of Minnesota | Programmable assembly of virus composites for receptor-targeted gene delivery |
| EP3717636B1 (en) | 2017-11-27 | 2023-03-08 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and use for inhibiting angiogenesis |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| WO2019152843A1 (en) | 2018-02-01 | 2019-08-08 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| EP3749754A4 (en) | 2018-02-08 | 2021-10-20 | Applied Stemcell, Inc. | TARGET GENE VARIANT SCREENING METHODS |
| US11306329B2 (en) | 2018-02-19 | 2022-04-19 | City Of Hope | Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof |
| WO2019178426A1 (en) | 2018-03-14 | 2019-09-19 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| EA202092362A1 (ru) | 2018-04-03 | 2021-01-11 | Страйдбайо, Инк. | Вирусные векторы для нацеливания на ткани глаза |
| CA3098565A1 (en) | 2018-04-29 | 2019-11-07 | Claire G. ZHANG | Scalable clarification process for recombinant aav production |
| EP3788165A1 (en) | 2018-04-29 | 2021-03-10 | REGENXBIO Inc. | Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles |
| CN112368391A (zh) | 2018-05-11 | 2021-02-12 | 马萨诸塞眼科耳科诊所 | 腺相关病毒的肝脏特异性嗜性 |
| CA3099306A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| US11821009B2 (en) | 2018-05-15 | 2023-11-21 | Cornell University | Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery |
| WO2019241535A2 (en) | 2018-06-14 | 2019-12-19 | Regenxbio Inc. | Anion exchange chromatography for recombinant aav production |
| WO2019240431A1 (ko) * | 2018-06-14 | 2019-12-19 | (주) 에빅스젠 | 세포 투과 펩티드 및 rpe65의 융합 단백질을 포함하는 레버 선천성 흑암시 치료용 약학적 조성물 |
| WO2020014471A1 (en) | 2018-07-11 | 2020-01-16 | The Brigham And Women's Hospital, Inc. | Methods and compositions for delivery of agents across the blood-brain barrier |
| CN112567027A (zh) | 2018-08-10 | 2021-03-26 | 再生生物股份有限公司 | 用于重组aav生产的可扩展方法 |
| WO2020068990A1 (en) | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CA3113975A1 (en) | 2018-10-01 | 2020-04-09 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| AU2019354995B2 (en) | 2018-10-02 | 2024-12-12 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| US20210324483A1 (en) | 2018-10-15 | 2021-10-21 | Regenxbio Inc. | Method for measuring the infectivity of replication defective viral vectors and viruses |
| CA3121177A1 (en) * | 2018-12-05 | 2020-06-11 | Abeona Therapeutics Inc. | Recombinant adeno-associated viral vector for gene delivery |
| WO2020123868A1 (en) * | 2018-12-12 | 2020-06-18 | Tesseract Health, Inc. | Biometric identification and health status determination |
| WO2020132578A1 (en) * | 2018-12-21 | 2020-06-25 | Chien Wade Wei De | Adeno-associated viruses and their uses for inner ear therapy |
| CN110437317B (zh) * | 2019-01-30 | 2023-05-02 | 上海科技大学 | 具有变异衣壳蛋白的腺相关病毒及其用途 |
| KR20210126096A (ko) | 2019-02-15 | 2021-10-19 | 상가모 테라퓨틱스, 인코포레이티드 | 재조합 aav를 제조하기 위한 조성물 및 방법 |
| PH12021552038A1 (en) | 2019-02-25 | 2023-01-04 | Friedrich Miescher Institute For Biomedical Res | Compositions and methods to treat bietti crystalline dystrophy |
| JP2022520875A (ja) | 2019-02-25 | 2022-04-01 | ノバルティス アーゲー | Biettiクリスタリン網膜症を治療するための組成物及び方法 |
| CN111621502B (zh) * | 2019-02-28 | 2023-05-02 | 武汉纽福斯生物科技有限公司 | 视网膜劈裂蛋白的编码序列、其表达载体构建及其应用 |
| JP2022522756A (ja) | 2019-02-28 | 2022-04-20 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 治療薬の送達のためのアデノ随伴ウイルスベクター |
| EP3934698A1 (en) | 2019-03-04 | 2022-01-12 | Adverum Biotechnologies, Inc. | Sequential intravitreal administration of aav gene therapy to contralateral eyes |
| EP3715358A1 (en) * | 2019-03-28 | 2020-09-30 | Universität zu Köln | Mutated adeno-associated virus capsid proteins, aav particle comprising the same and liver directed aav vector gene therapy |
| JP7630176B2 (ja) | 2019-04-01 | 2025-02-17 | テナヤ セラピューティクス, インコーポレイテッド | 操作されたカプシドを有するアデノ随伴ウイルス |
| JP2022521851A (ja) | 2019-04-03 | 2022-04-12 | レジェンクスバイオ インコーポレーテッド | 眼の病態に対する遺伝子療法 |
| TW202102526A (zh) * | 2019-04-04 | 2021-01-16 | 美商銳進科斯生物股份有限公司 | 重組腺相關病毒及其用途 |
| HUE064411T2 (hu) | 2019-04-11 | 2024-03-28 | Regenxbio Inc | Méretkizárásos kromatográfiás módszerek rekombináns adeno-asszociált víruskészítmények jellemzésére |
| MX2021012564A (es) | 2019-04-19 | 2022-01-18 | Regenxbio Inc | Formulaciones y métodos de vectores de virus adenoasociados. |
| KR20220004072A (ko) | 2019-04-24 | 2022-01-11 | 다카라 바이오 가부시키가이샤 | 뇌에 지향성을 가지는 aav 돌연변이체 |
| CN114144197A (zh) | 2019-04-24 | 2022-03-04 | 再生生物股份有限公司 | 完全人类翻译后修饰的抗体治疗剂 |
| TWI862583B (zh) * | 2019-04-26 | 2024-11-21 | 美商愛德維仁生物科技公司 | 用於玻璃體內遞送之變異體aav蛋白殼 |
| JP2022530845A (ja) * | 2019-04-27 | 2022-07-04 | オクジェン アイエヌシー. | 眼疾患に対するadeno随伴ウイルス連ウイルスベクター媒介遺伝子治療 |
| US11737665B2 (en) | 2019-06-21 | 2023-08-29 | Tesseract Health, Inc. | Multi-modal eye imaging with shared optical path |
| EP3989982A4 (en) * | 2019-06-27 | 2023-06-28 | University Of Florida Research Foundation, Incorporated | Enhancing aav-mediated transduction of ocular tissues with hyaluronic acid |
| WO2021005223A1 (en) | 2019-07-10 | 2021-01-14 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for the treatment of epilepsy |
| ES2964907T3 (es) * | 2019-07-11 | 2024-04-10 | Centre Nat Rech Scient | Virus adenoasociado modificado químicamente |
| US10557149B1 (en) | 2019-07-15 | 2020-02-11 | Vigene Biosciences, Inc. | Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus |
| US10653731B1 (en) | 2019-07-15 | 2020-05-19 | Vigene Biosciences Inc. | Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency |
| US10801042B1 (en) | 2019-07-15 | 2020-10-13 | Vigene Biosciences, Inc. | Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus |
| US20230042103A1 (en) | 2019-07-26 | 2023-02-09 | Regenxbio Inc. | Engineered nucleic acid regulatory element and methods of uses thereof |
| RU2751592C2 (ru) | 2019-08-22 | 2021-07-15 | Общество С Ограниченной Ответственностью "Анабион" | Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе |
| KR20220051246A (ko) | 2019-08-26 | 2022-04-26 | 리젠엑스바이오 인크. | 완전 인간 번역 후 변형된 항-VEGF Fab를 사용한 당뇨병성 망막병증의 치료 |
| MX2022002961A (es) | 2019-09-11 | 2022-04-06 | Adverum Biotechnologies Inc | Metodos para tratar enfermedades neovasculares oculares usando variantes de aav2 que codifican para aflibercept. |
| US20220325296A1 (en) * | 2019-09-12 | 2022-10-13 | The Broad Institute, Inc. | Engineered adeno-associated virus capsids |
| GB201913974D0 (en) | 2019-09-27 | 2019-11-13 | King S College London | Vector |
| JP2022550435A (ja) | 2019-10-04 | 2022-12-01 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | 組換えaavの改善された治療的使用のための方法 |
| JP2022552262A (ja) | 2019-10-07 | 2022-12-15 | リジェネックスバイオ インコーポレイテッド | アデノ随伴ウイルスベクター医薬組成物および方法 |
| WO2021077000A1 (en) | 2019-10-16 | 2021-04-22 | The Broad Institute, Inc. | Engineered muscle targeting compositions |
| EP4045664A1 (en) * | 2019-10-16 | 2022-08-24 | Wuxi Apptec (Shanghai) Co., Ltd. | A novel aav variant |
| CA3157464A1 (en) * | 2019-10-31 | 2021-05-06 | Universitat Bern | Aav vector variants for ocular gene delivery |
| AU2020379046B2 (en) * | 2019-11-08 | 2025-03-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for X-linked juvenile retinoschisis therapy |
| WO2021099394A1 (en) | 2019-11-19 | 2021-05-27 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Antisense oligonucleotides and their use for the treatment of cancer |
| CN115335529A (zh) * | 2019-11-26 | 2022-11-11 | 马萨诸塞大学 | 用于递送kh902(康柏西普)的重组腺相关病毒及其用途 |
| JP2023503637A (ja) | 2019-11-28 | 2023-01-31 | リジェネックスバイオ インコーポレイテッド | マイクロジストロフィン遺伝子治療コンストラクト及びその使用 |
| US11981705B2 (en) | 2020-01-10 | 2024-05-14 | The Brigham And Women's Hospital, Inc. | Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| EP4093428A1 (en) | 2020-01-22 | 2022-11-30 | RegenxBio Inc. | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
| AR121201A1 (es) | 2020-01-29 | 2022-04-27 | Regenxbio Inc | Tratamiento de mucopolisacaridosis iva |
| US20230088992A1 (en) | 2020-01-29 | 2023-03-23 | Regenxbio Inc. | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells |
| MX2022009982A (es) | 2020-02-14 | 2022-09-12 | Ultragenyx Pharmaceutical Inc | Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5. |
| EP4121544A1 (en) | 2020-03-19 | 2023-01-25 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| EP4127189A1 (en) | 2020-03-31 | 2023-02-08 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| US20230159609A1 (en) * | 2020-04-10 | 2023-05-25 | Sorbonne Université | G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd) |
| US20230190956A1 (en) | 2020-04-24 | 2023-06-22 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for preventing induction of immune responses to the transduced cells expressing a transgene product after ocular gene therapy |
| MX2022013411A (es) | 2020-04-27 | 2022-11-14 | 4D Molecular Therapeutics Inc | Genes gla con codones optimizados y usos de los mismos. |
| PE20230767A1 (es) | 2020-05-13 | 2023-05-09 | Voyager Therapeutics Inc | Redireccion del tropismo de las capsides de aav |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復pah基因功能的腺相關病毒組成物及其使用方法 |
| MX2022015048A (es) | 2020-05-29 | 2023-03-08 | Univ California | Viriones de virus adenoasociados con variantes de capsides y metodos de uso de estos. |
| CA3177613A1 (en) | 2020-07-10 | 2022-04-13 | Inserm (Institut National De La Sante Et De La Recherche Medicale) | Methods and compositions for treating epilepsy |
| CN112226461B (zh) * | 2020-08-21 | 2022-04-22 | 华侨大学 | Cd4阳性细胞特异性基因传递载体及其应用 |
| JP2023538129A (ja) * | 2020-08-21 | 2023-09-06 | カプシダ, インコーポレイテッド | 好ましい発現レベルを有するアデノ随伴ウイルス組成物 |
| US12129287B2 (en) | 2020-09-14 | 2024-10-29 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| EP4213890A1 (en) | 2020-09-15 | 2023-07-26 | RegenxBio Inc. | Vectorized lanadelumab and administration thereof |
| WO2022060916A1 (en) | 2020-09-15 | 2022-03-24 | Regenxbio Inc. | Vectorized antibodies for anti-viral therapy |
| CN113717248B (zh) * | 2020-09-30 | 2022-07-08 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2022076750A2 (en) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns or muscle delivery |
| CA3198368A1 (en) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Formulations for suprachoroidal administration such as formulations with aggregate formation |
| JP2023545722A (ja) | 2020-10-07 | 2023-10-31 | リジェネックスバイオ インコーポレイテッド | 遺伝子治療剤の眼送達のためのアデノ随伴ウイルス |
| TW202228646A (zh) | 2020-10-07 | 2022-08-01 | 美商銳進科斯生物股份有限公司 | 諸如凝膠調配物之用於脈絡膜上投與之調配物 |
| JP2023544797A (ja) | 2020-10-07 | 2023-10-25 | レジェンクスバイオ インコーポレーテッド | 脈絡膜上投与用の、高粘度製剤などの製剤 |
| WO2022094157A1 (en) | 2020-10-28 | 2022-05-05 | Regenxbio Inc. | Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof |
| AR123948A1 (es) | 2020-10-28 | 2023-01-25 | Regenxbio Inc | ANTICUERPOS ANTI-TNF-a VECTORIZADOS PARA INDICACIONES OCULARES |
| IL302282A (en) | 2020-10-29 | 2023-06-01 | Regenxbio Inc | Vectorized tnf-alpha antagonists for ocular indications |
| WO2022094255A2 (en) | 2020-10-29 | 2022-05-05 | Regenxbio Inc. | Vectorized factor xii antibodies and administration thereof |
| KR20230107285A (ko) * | 2020-11-10 | 2023-07-14 | 에이버맥스 바이오파마 인크. | 조작된 바이러스 캡시드 및 사용 방법 |
| US20240002839A1 (en) | 2020-12-02 | 2024-01-04 | Decibel Therapeutics, Inc. | Crispr sam biosensor cell lines and methods of use thereof |
| US20240067678A1 (en) * | 2020-12-16 | 2024-02-29 | Children's Medical Research Institute | Adeno-associated virus capsids and vectors |
| IL303614A (en) | 2020-12-16 | 2023-08-01 | Regenxbio Inc | Method of producing a recombinant adeno-associated virus particle |
| WO2022147087A1 (en) | 2020-12-29 | 2022-07-07 | Regenxbio Inc. | Tau-specific antibody gene therapy compositions, methods and uses thereof |
| WO2022159662A1 (en) | 2021-01-21 | 2022-07-28 | Regenxbio Inc. | Improved production of recombinant polypeptides and viruses |
| JP2024505739A (ja) | 2021-02-10 | 2024-02-07 | レジェンクスバイオ インコーポレーテッド | 組換えヒトイズロン酸-2-スルファターゼ(ids)によるムコ多糖症iiの治療 |
| CN113121651B (zh) * | 2021-04-19 | 2023-11-17 | 信念医药科技(上海)有限公司 | 低中和抗体腺相关病毒衣壳蛋白 |
| CN113121652B (zh) * | 2021-04-19 | 2022-10-11 | 上海信致医药科技有限公司 | 视网膜和肌肉高亲和性腺相关病毒衣壳蛋白及相关应用 |
| EP4326339B1 (en) | 2021-04-20 | 2025-04-02 | Institut National de la Santé et de la Recherche Médicale (INSERM) | Compositions and methods for treating retinal degenerative disorders |
| JP2024517143A (ja) | 2021-04-26 | 2024-04-19 | リジェネックスバイオ インコーポレイテッド | ジストロフィン異常症の治療のためのマイクロジストロフィン遺伝子療法投与 |
| JP2024515459A (ja) | 2021-04-27 | 2024-04-10 | アドヴェラム バイオテクノロジーズ, インコーポレイテッド | アフリベルセプトをコードするaav2バリアントを使用する眼疾患の治療方法 |
| WO2022235614A2 (en) | 2021-05-04 | 2022-11-10 | Regenxbio Inc. | Novel aav vectors and methods and uses thereof |
| EP4337267A1 (en) | 2021-05-11 | 2024-03-20 | RegenxBio Inc. | Treatment of duchenne muscular dystrophy and combinations thereof |
| WO2022248634A1 (en) | 2021-05-26 | 2022-12-01 | Sparingvision | G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd) |
| JP2024519888A (ja) * | 2021-05-28 | 2024-05-21 | 上海瑞宏迪医薬有限公司 | カプシド変異を有する組換えアデノ随伴ウイルス及びその応用 |
| AU2022283898A1 (en) * | 2021-06-03 | 2024-01-04 | Dyno Therapeutics, Inc. | Capsid variants and methods of using the same |
| EP4366757A1 (en) | 2021-07-07 | 2024-05-15 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Synergistic combination of rdcfv2 and rdcvf2l for the treatment of tauopathies |
| AU2022307365A1 (en) | 2021-07-08 | 2024-02-01 | Tenaya Therapeutics, Inc. | Optimized expression cassettes for gene therapy |
| EP4373947A1 (en) | 2021-07-19 | 2024-05-29 | New York University | Auf1 combination therapies for treatment of muscle degenerative disease |
| US20250019721A1 (en) | 2021-08-11 | 2025-01-16 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating a muscular dystrophy |
| AR126840A1 (es) | 2021-08-20 | 2023-11-22 | Llc «Anabion» | Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en esta |
| MX2024002200A (es) | 2021-08-20 | 2024-04-29 | Biocad Joint Stock Co | Metodo para obtener una capside de virus adenoasociado modificada. |
| AR126839A1 (es) | 2021-08-20 | 2023-11-22 | Llc «Anabion» | Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta |
| CN118202060A (zh) | 2021-10-05 | 2024-06-14 | 再生生物股份有限公司 | 用于重组aav生产的组合物和方法 |
| WO2023060113A1 (en) | 2021-10-05 | 2023-04-13 | Regenxbio Inc. | Compositions and methods for recombinant aav production |
| WO2023060269A1 (en) | 2021-10-07 | 2023-04-13 | Regenxbio Inc. | Recombinant adeno-associated viruses for targeted delivery |
| WO2023060272A2 (en) | 2021-10-07 | 2023-04-13 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns tropic delivery |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| EP4163296A1 (en) | 2021-10-11 | 2023-04-12 | Sparingvision | G-protein-gated-k+ channel-mediated enhancements in light sensitivity in patients with rod-cone dystrophy (rcd) |
| EP4423285A1 (en) | 2021-10-28 | 2024-09-04 | RegenxBio Inc. | Engineered nucleic acid regulatory elements and methods and uses thereof |
| IL311861A (en) | 2021-11-02 | 2024-06-01 | Voyager Therapeutics Inc | Aav capsid variants and uses thereof |
| WO2023108507A1 (en) * | 2021-12-15 | 2023-06-22 | National Institute Of Biological Sciences, Beijing | Recombinant aav vectors and use thereof |
| US20250137011A1 (en) | 2022-02-02 | 2025-05-01 | Adverum Biotechnologies, Inc. | Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept |
| CA3244250A1 (en) | 2022-02-16 | 2023-08-24 | Adverum Biotechnologies, Inc. | METHOD FOR REDUCING CST FLUCTUATION IN NEOVASULAR AMD BY A RECOMBINANT ADENO-ASSOCIATED VIRUS |
| KR20240152351A (ko) * | 2022-02-17 | 2024-10-21 | 스카이라인 테라퓨틱스 리미티드 | 변형된 aav 캡시드 폴리펩티드를 갖는 재조합 아데노-연관 바이러스 |
| CA3245327A1 (en) | 2022-03-07 | 2023-09-14 | Ultragenyx Pharmaceutical Inc. | MODIFIED BATCH-BASED AAV PRODUCTION SYSTEMS AND PROCESSES |
| WO2023178053A1 (en) | 2022-03-13 | 2023-09-21 | Regenxbio Inc. | Modified muscle-specific promoters |
| EP4499154A1 (en) | 2022-03-25 | 2025-02-05 | REGENXBIO Inc. | Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy |
| EP4504149A1 (en) | 2022-04-06 | 2025-02-12 | RegenxBio Inc. | Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene for suprachoidal administration |
| TW202345913A (zh) | 2022-04-06 | 2023-12-01 | 美商銳進科斯生物股份有限公司 | 用於脈絡膜上投與之調配物諸如凝膠調配物 |
| TW202404651A (zh) | 2022-04-06 | 2024-02-01 | 美商銳進科斯生物股份有限公司 | 用於脈絡膜上投與之調配物諸如形成聚集體之調配物 |
| EP4508062A1 (en) | 2022-04-11 | 2025-02-19 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| CN120590489A (zh) * | 2022-04-12 | 2025-09-05 | 合肥星眸生物科技有限公司 | 一种融合型腺相关病毒及其应用 |
| WO2023201277A1 (en) | 2022-04-14 | 2023-10-19 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns tropic delivery |
| WO2023201308A1 (en) | 2022-04-14 | 2023-10-19 | Regenxbio Inc. | Gene therapy for treating an ocular disease |
| CN116970648A (zh) * | 2022-04-24 | 2023-10-31 | 上海朗昇生物科技有限公司 | 新型aav衣壳改造株及其用途 |
| US20250288697A1 (en) | 2022-05-03 | 2025-09-18 | Regenxbio Inc. | Vectorized anti-tnf-alpha inhibitors for ocular indications |
| WO2023215806A2 (en) | 2022-05-03 | 2023-11-09 | Regenxbio Inc. | Vectorized anti-complement antibodies and complement agents and administration thereof |
| KR20250022114A (ko) | 2022-06-07 | 2025-02-14 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 시력 회복을 위한 멜라놉신 변이체 |
| WO2023239627A2 (en) | 2022-06-08 | 2023-12-14 | Regenxbio Inc. | Methods for recombinant aav production |
| WO2024017990A1 (en) | 2022-07-21 | 2024-01-25 | Institut National de la Santé et de la Recherche Médicale | Methods and compositions for treating chronic pain disorders |
| CA3264522A1 (en) | 2022-08-11 | 2024-02-15 | Institute Of Molecular And Clinical Ophthalmology Basel (Iob) | OPTOGENIC CONSTRUCTIONS OF HUMAN CONICAL PHOTORECEPTORS |
| JP2025527658A (ja) | 2022-08-24 | 2025-08-22 | リジェネックスバイオ インコーポレイテッド | 組換えアデノ随伴ウイルス及びその使用 |
| KR20250099772A (ko) | 2022-09-30 | 2025-07-02 | 리젠엑스바이오 인크. | 항-vegf fab를 암호화하는 재조합 바이러스 벡터를 이용한 안구 질환의 치료 |
| WO2024081746A2 (en) | 2022-10-11 | 2024-04-18 | Regenxbio Inc. | Engineered nucleic acid regulatory elements and methods and uses thereof |
| IL320351A (en) | 2022-10-20 | 2025-06-01 | Sparingvision | Compositions and methods for treating retinal degenerative disorders |
| EP4357359A1 (en) | 2022-10-20 | 2024-04-24 | Sparingvision | G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd) |
| CN120112547A (zh) | 2022-11-16 | 2025-06-06 | 瑞泽恩制药公司 | 包含膜结合il-12和蛋白酶可裂解接头的嵌合蛋白 |
| CN120752339A (zh) | 2023-01-06 | 2025-10-03 | 国家医疗保健研究所 | 静脉内施用用于治疗疼痛的反义寡核苷酸 |
| CN118359687A (zh) * | 2023-01-18 | 2024-07-19 | 上海朗昇生物科技有限公司 | 一种新衣壳蛋白变体以及其用途 |
| WO2024161142A1 (en) | 2023-02-02 | 2024-08-08 | Quethera Limited | Recombinant adeno-associated virus vector |
| WO2024191778A1 (en) | 2023-03-10 | 2024-09-19 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2024192281A2 (en) | 2023-03-15 | 2024-09-19 | Regenxbio Inc. | Exon skipping gene therapy constructs, vectors and uses thereof |
| WO2024211831A1 (en) | 2023-04-05 | 2024-10-10 | Adverum Biotechnologies, Inc. | Immune landscape signatures for ocular inflammation |
| WO2024211780A1 (en) | 2023-04-07 | 2024-10-10 | Regenxbio Inc. | Compositions and methods for recombinant aav production |
| WO2024215653A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Guide rnas, vectors, and virions for targeting mutations in the pln gene |
| WO2024215655A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Cardioprotective bag3 therapies |
| WO2024216244A2 (en) | 2023-04-13 | 2024-10-17 | Regenxbio Inc. | Targeting aav capsids, methods of manufacturing and using same |
| WO2024229389A1 (en) * | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of tau-related disorders |
| TW202444917A (zh) * | 2023-05-04 | 2024-11-16 | 美商航海家醫療公司 | Aav衣殼變異體及其用途 |
| WO2024233529A2 (en) | 2023-05-07 | 2024-11-14 | Regenxbio Inc. | Compositions and methods for recombinant aav production |
| WO2024238859A1 (en) | 2023-05-16 | 2024-11-21 | Regenxbio Inc. | Vectorized c5 inhibitor agents and administration thereof |
| WO2024238867A1 (en) | 2023-05-16 | 2024-11-21 | Regenxbio Inc. | Vectorized anti-complement antibodies and administration thereof |
| WO2024238853A1 (en) | 2023-05-16 | 2024-11-21 | Regenxbio Inc. | Adeno-associated viruses for ocular delivery of gene therapy |
| WO2024240113A1 (zh) * | 2023-05-23 | 2024-11-28 | 北京因诺惟康医药科技有限公司 | 一种腺相关病毒衣壳蛋白、包含其的腺相关病毒、载体及应用 |
| WO2025008406A1 (en) | 2023-07-04 | 2025-01-09 | Institut National de la Santé et de la Recherche Médicale | Antisense oligonucleotides and their use for the treatment of cancer |
| WO2025015317A2 (en) | 2023-07-13 | 2025-01-16 | Kriya Therapeutics, Inc. | Complement pathway inhibitor fusions and methods of using the same |
| CN117230117A (zh) * | 2023-08-04 | 2023-12-15 | 中国科学院深圳先进技术研究院 | 重组腺相关病毒包装质粒、突变体及其制备方法和应用 |
| WO2025049993A1 (en) | 2023-08-31 | 2025-03-06 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2025075963A1 (en) | 2023-10-02 | 2025-04-10 | Regenxbio Inc. | Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase |
| WO2025090962A1 (en) | 2023-10-25 | 2025-05-01 | Regenxbio Inc. | Compositions and methods for recombinant aav production |
| CN117247434B (zh) * | 2023-11-10 | 2024-02-02 | 上海朗昇生物科技有限公司 | 衣壳修饰型病毒载体及其制备和用途 |
| WO2025106374A1 (en) | 2023-11-13 | 2025-05-22 | Juno Therapeutics, Inc. | Aav production method |
| WO2025108407A2 (en) | 2023-11-23 | 2025-05-30 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Gene therapy compositions and methods for treating glioma |
| WO2025113676A1 (en) | 2023-11-29 | 2025-06-05 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treating stroke in primates |
| WO2025129085A1 (en) | 2023-12-15 | 2025-06-19 | Port Therapeutics, Inc. | Use of thermal bioswitches in aav-based gene therapy |
| CN119080889B (zh) * | 2024-09-18 | 2025-06-24 | 广州译码基因科技有限公司 | 提高aav对眼组织感染能力的衣壳蛋白突变体及应用 |
| WO2025171227A1 (en) | 2024-02-08 | 2025-08-14 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2025217230A1 (en) | 2024-04-08 | 2025-10-16 | Regenxbio Inc. | Vectorized anti-complement antibodies and complement agents and administration thereof |
| WO2025217214A2 (en) | 2024-04-08 | 2025-10-16 | Regenxbio Inc. | Recombinant adeno-associated viruses and uses thereof |
| WO2025214404A1 (en) | 2024-04-10 | 2025-10-16 | Skyline Therapeutics (Shanghai) Co., Ltd. | Optogenetic modulation for vision restoration |
| WO2025214408A1 (en) | 2024-04-10 | 2025-10-16 | Skyline Therapeutics (Shanghai) Co., Ltd. | Optogenetic modulation for vision restoration |
| WO2025237990A1 (en) | 2024-05-14 | 2025-11-20 | Institut National de la Santé et de la Recherche Médicale | Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis |
| CN118955654B (zh) * | 2024-10-15 | 2025-05-16 | 朗信启昇(苏州)生物制药有限公司 | 具有变异衣壳的腺相关病毒病毒体及其用途 |
Family Cites Families (103)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB9206016D0 (en) | 1992-03-19 | 1992-04-29 | Sandoz Ltd | Improvements in or relating to organic compounds |
| US5639872A (en) | 1993-07-27 | 1997-06-17 | Hybridon, Inc. | Human VEGF-specific oligonucleotides |
| US6440425B1 (en) | 1995-05-01 | 2002-08-27 | Aventis Pasteur Limited | High molecular weight major outer membrane protein of moraxella |
| US6001650A (en) * | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| AU1686997A (en) | 1995-12-08 | 1997-06-27 | Hybridon, Inc. | Modified vegf antisense oligonucleotides for treatment of skin disorders |
| US6096548A (en) | 1996-03-25 | 2000-08-01 | Maxygen, Inc. | Method for directing evolution of a virus |
| CA2251738A1 (en) | 1996-04-16 | 1997-10-23 | Immusol Incorporated | Targeted viral vectors |
| WO1998011244A2 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6710036B2 (en) | 1997-07-25 | 2004-03-23 | Avigen, Inc. | Induction of immune response to antigens expressed by recombinant adeno-associated virus |
| AU9319198A (en) | 1997-09-19 | 1999-04-05 | Trustees Of The University Of Pennsylvania, The | Methods and vector constructs useful for production of recombinant aav |
| EP1690868A1 (en) | 1997-10-31 | 2006-08-16 | Maxygen, Inc. | Modification of virus tropism and host range by viral genome shuffling |
| US6410300B1 (en) | 1998-01-12 | 2002-06-25 | The University Of North Carolina At Chapel Hill | Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV |
| US6551795B1 (en) * | 1998-02-18 | 2003-04-22 | Genome Therapeutics Corporation | Nucleic acid and amino acid sequences relating to pseudomonas aeruginosa for diagnostics and therapeutics |
| ES2313784T3 (es) | 1998-05-28 | 2009-03-01 | The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services | Vector aav5 y usos del mismo. |
| DE19827457C1 (de) | 1998-06-19 | 2000-03-02 | Medigene Ag | Strukturprotein von AAV, seine Herstellung und Verwendung |
| US6451594B1 (en) * | 1998-09-11 | 2002-09-17 | The Regents Of The University Of California | Recombinant adenovirus for tissue specific expression in heart |
| EP1127150B1 (en) | 1998-11-05 | 2007-05-16 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| WO2000028004A1 (en) | 1998-11-10 | 2000-05-18 | The University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
| US6943153B1 (en) | 1999-03-15 | 2005-09-13 | The Regents Of The University Of California | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
| US6498244B1 (en) | 1999-05-28 | 2002-12-24 | Cell Genesys, Inc. | Adeno-associated virus capsid immunologic determinants |
| US7314912B1 (en) | 1999-06-21 | 2008-01-01 | Medigene Aktiengesellschaft | AAv scleroprotein, production and use thereof |
| DE19933288A1 (de) | 1999-07-15 | 2001-01-18 | Medigene Ag | Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung |
| DE19933719A1 (de) | 1999-07-19 | 2001-01-25 | Medigene Ag | Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung |
| US8232081B2 (en) | 1999-09-21 | 2012-07-31 | Basf Se | Methods and microorganisms for production of panto-compounds |
| DE10001363A1 (de) | 2000-01-14 | 2001-07-26 | Windmoeller & Hoelscher | Extruderdüsenkopf |
| US20070026394A1 (en) | 2000-02-11 | 2007-02-01 | Lawrence Blatt | Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies |
| US6855314B1 (en) | 2000-03-22 | 2005-02-15 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector for transducing brain cells and lung cells |
| JP2004524813A (ja) * | 2000-09-22 | 2004-08-19 | バイレクシス コーポレイション | 改良された条件付けで複製するベクター類、それらの生成及び使用方法 |
| AU2002248297A1 (en) * | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| US7749492B2 (en) * | 2001-01-05 | 2010-07-06 | Nationwide Children's Hospital, Inc. | AAV vectors and methods |
| US20030144221A1 (en) | 2001-07-17 | 2003-07-31 | Isis Pharmaceuticals Inc. | Antisense modulation of BCL2-associated X protein expression |
| US7647184B2 (en) | 2001-08-27 | 2010-01-12 | Hanall Pharmaceuticals, Co. Ltd | High throughput directed evolution by rational mutagenesis |
| US20030129203A1 (en) * | 2001-08-27 | 2003-07-10 | Nautilus Biotech S.A. | Mutant recombinant adeno-associated viruses |
| WO2003042361A2 (en) | 2001-11-09 | 2003-05-22 | Government Of The United States Of America, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| MX346493B (es) | 2001-11-13 | 2017-03-21 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| US20050106558A1 (en) | 2001-12-21 | 2005-05-19 | Luca Perabo | Library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism |
| DE10216005A1 (de) | 2002-04-11 | 2003-10-30 | Max Planck Gesellschaft | Verwendung von biologischen Photorezeptoren als direkt lichtgesteuerte Ionenkanäle |
| WO2003089612A2 (en) | 2002-04-17 | 2003-10-30 | University Of Florida Research Foundation, Inc. | IMPROVED rAAV VECTORS |
| AU2003223775A1 (en) | 2002-04-30 | 2003-11-17 | Duke University | Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use |
| WO2003093436A2 (en) | 2002-04-30 | 2003-11-13 | University Of Florida | Treatment for phenylketonuria |
| US7254489B2 (en) | 2002-05-31 | 2007-08-07 | Microsoft Corporation | Systems, methods and apparatus for reconstructing phylogentic trees |
| US7148342B2 (en) | 2002-07-24 | 2006-12-12 | The Trustees Of The University Of Pennyslvania | Compositions and methods for sirna inhibition of angiogenesis |
| WO2004020600A2 (en) | 2002-08-28 | 2004-03-11 | University Of Florida | Modified aav |
| EP1576001A2 (en) * | 2002-12-18 | 2005-09-21 | Novartis AG | Endothelial cell specifically binding peptides |
| US20070172460A1 (en) | 2003-03-19 | 2007-07-26 | Jurgen Kleinschmidt | Random peptide library displayed on aav vectors |
| WO2004108922A2 (en) | 2003-04-25 | 2004-12-16 | The Trustees Of The University Of Pennsylvania | Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver |
| AU2004238979B2 (en) * | 2003-05-14 | 2008-12-18 | Istituto Di Ricerche Di Biologia Molecolare P Angeletti Spa | Broadening adenovirus tropism |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| WO2004112727A2 (en) | 2003-06-19 | 2004-12-29 | Avigen, Inc. | Aav virions with decreased immunoreactivity and uses therefor |
| EP1644038A2 (en) | 2003-06-23 | 2006-04-12 | A & G Pharmaceuticals, Inc. | Compositions and methods for restoring sensitivity of tumor cells to antitumor therapy and inducing apoptosis |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US20050148069A1 (en) | 2003-09-24 | 2005-07-07 | Gage Fred H. | IGF-1 instructs multipotent adult CNS neural stem cells to an oligodendroglial lineage |
| CN102199626B (zh) | 2003-09-30 | 2015-06-24 | 宾夕法尼亚大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| KR100550967B1 (ko) | 2003-12-03 | 2006-02-13 | 주식회사 엘지생활건강 | 유중수형 화장료 조성물 |
| EP2365077B1 (en) | 2004-03-12 | 2013-05-08 | Alnylam Pharmaceuticals, Inc. | iRNA agents targeting VEGF |
| US7427396B2 (en) | 2004-06-03 | 2008-09-23 | Genzyme Corporation | AAV vectors for gene delivery to the lung |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| US20060211650A1 (en) | 2004-12-16 | 2006-09-21 | Forest Laboratories, Inc. | Reducing carbohydrate derivatives of adamantane amines, and synthesis and methods of use thereof |
| US20100172871A1 (en) | 2005-02-17 | 2010-07-08 | Flannery John G | Muller Cell Specific Gene Therapy |
| EP2383346B1 (en) | 2005-04-07 | 2014-10-08 | The Trustees of the University of Pennsylvania | Modified AAVrh.64 capsids, compositions containing same and uses thereof |
| AU2006284425A1 (en) | 2005-07-22 | 2007-03-01 | The Board Of Trustees Of The Leland Stanford Junior University | Light-activated cation channel and uses thereof |
| CN1834255A (zh) * | 2005-12-23 | 2006-09-20 | 上海交通大学附属第一人民医院 | 加快并提高腺相关病毒介导的基因在视网膜细胞内表达的方法 |
| US7867484B2 (en) | 2006-01-27 | 2011-01-11 | University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| EP3437473A1 (en) * | 2006-05-04 | 2019-02-06 | Wayne State University | Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids |
| ES2785223T3 (es) * | 2006-06-21 | 2020-10-06 | Uniqure Ip Bv | Células de insecto para la producción de vectores de AAV |
| US7872118B2 (en) | 2006-09-08 | 2011-01-18 | Opko Ophthalmics, Llc | siRNA and methods of manufacture |
| CN1966082B (zh) * | 2006-11-03 | 2010-06-30 | 许瑞安 | 一种防治结直肠癌的基因药物及其制备方法和用途 |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| NZ593611A (en) | 2007-04-13 | 2012-06-29 | Catalyst Biosciences Inc | Modified factor vii polypeptides and uses thereof |
| EP1985708B1 (en) * | 2007-04-27 | 2015-04-15 | Universität Rostock | Selective targeting of viruses to neural precursor cells |
| US8632764B2 (en) | 2008-04-30 | 2014-01-21 | University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| AU2009274482A1 (en) | 2008-05-20 | 2010-01-28 | Eos Neuroscience, Inc. | Vectors for delivery of light-sensitive proteins and methods of use |
| EP2297185A1 (en) | 2008-06-17 | 2011-03-23 | Amsterdam Molecular Therapeutics (AMT) B.V. | Parvoviral capsid with incorporated gly-ala repeat region |
| EP2396343B1 (en) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| CN101532024A (zh) * | 2009-04-30 | 2009-09-16 | 许瑞安 | 一种用于基因治疗的新型细胞特异性内含microRNA结合序列的基因HAAVmir |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| WO2011117258A2 (en) | 2010-03-22 | 2011-09-29 | Association Institut De Myologie | Methods of increasing efficiency of vector penetration of target tissue |
| EP2571512B1 (en) | 2010-05-17 | 2017-08-23 | Sangamo BioSciences, Inc. | Novel dna-binding proteins and uses thereof |
| US8663624B2 (en) * | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| WO2012109570A1 (en) * | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| JP6072772B2 (ja) | 2011-04-22 | 2017-02-01 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法 |
| EP3795581A3 (en) | 2011-08-24 | 2021-06-09 | The Board of Trustees of the Leland Stanford Junior University | New avv capsid proteins for nucleic acid transfer |
| JP6385920B2 (ja) | 2012-05-09 | 2018-09-05 | オレゴン ヘルス アンド サイエンス ユニバーシティー | アデノ随伴ウイルスプラスミド及びベクター |
| TWI702955B (zh) | 2012-05-15 | 2020-09-01 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| WO2014103957A1 (ja) | 2012-12-25 | 2014-07-03 | タカラバイオ株式会社 | Aav変異体 |
| DK2954051T3 (da) | 2013-02-08 | 2019-07-08 | Univ Pennsylvania | Modificeret kapsid til genoverførsel til behandling af nethinden |
| EP3004339B1 (en) | 2013-05-29 | 2021-07-07 | Cellectis | New compact scaffold of cas9 in the type ii crispr system |
| WO2014194132A1 (en) | 2013-05-31 | 2014-12-04 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| AU2014324717B2 (en) | 2013-09-26 | 2020-10-08 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial AAV capsid library for targeted gene therapy |
| PL3459965T3 (pl) | 2013-10-11 | 2021-07-26 | Massachusetts Eye & Ear Infirmary | Sposoby przewidywania sekwencji wirusa-przodka i ich zastosowania |
| AU2015231439B2 (en) | 2014-03-17 | 2019-11-14 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| IL248102B (en) | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| CA2951707A1 (en) | 2014-06-10 | 2015-12-17 | Massachusetts Institute Of Technology | Method for gene editing |
| MY187898A (en) | 2015-03-02 | 2021-10-27 | Adverum Biotechnologies Inc | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| EP3748007B1 (en) | 2015-03-06 | 2024-06-26 | Massachusetts Eye & Ear Infirmary | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
| WO2017023724A1 (en) | 2015-07-31 | 2017-02-09 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of aadc deficiency |
| US11364308B2 (en) | 2016-05-13 | 2022-06-21 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and methods of use thereof |
| MA44546B1 (fr) * | 2016-06-15 | 2021-03-31 | Univ California | Virus adéno-associés variants et procédés d'utilisation |
| US20200121746A1 (en) * | 2017-06-30 | 2020-04-23 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsids and methods of use thereof |
| CA3059995A1 (en) | 2017-08-28 | 2019-03-07 | The Regents Of The University Of California | Adeno-associated virus capsid variants and methods of use thereof |
-
2012
- 2012-04-20 JP JP2014506568A patent/JP6072772B2/ja active Active
- 2012-04-20 PL PL21201945.9T patent/PL4005603T3/pl unknown
- 2012-04-20 LT LTEP17168534.0T patent/LT3254703T/lt unknown
- 2012-04-20 LT LTEP12774323.5T patent/LT2699270T/lt unknown
- 2012-04-20 MX MX2013012384A patent/MX349138B/es active IP Right Grant
- 2012-04-20 PT PT212019459T patent/PT4005603T/pt unknown
- 2012-04-20 SM SM20240520T patent/SMT202400520T1/it unknown
- 2012-04-20 CN CN201280024852.3A patent/CN103561774B/zh active Active
- 2012-04-20 PT PT201514791T patent/PT3693025T/pt unknown
- 2012-04-20 SG SG10201800873WA patent/SG10201800873WA/en unknown
- 2012-04-20 ES ES12774323.5T patent/ES2638342T3/es active Active
- 2012-04-20 HR HRP20241577TT patent/HRP20241577T1/hr unknown
- 2012-04-20 ES ES20151479T patent/ES2904638T3/es active Active
- 2012-04-20 ES ES17168534T patent/ES2791499T3/es active Active
- 2012-04-20 SI SI201232080T patent/SI4005603T1/sl unknown
- 2012-04-20 SM SM20200386T patent/SMT202000386T1/it unknown
- 2012-04-20 SI SI201231780T patent/SI3254703T1/sl unknown
- 2012-04-20 AU AU2012245328A patent/AU2012245328B2/en active Active
- 2012-04-20 IL IL280819A patent/IL280819B2/en unknown
- 2012-04-20 SM SM20170443T patent/SMT201700443T1/it unknown
- 2012-04-20 PT PT127743235T patent/PT2699270T/pt unknown
- 2012-04-20 SI SI201231974T patent/SI3693025T1/sl unknown
- 2012-04-20 PL PL20151479T patent/PL3693025T3/pl unknown
- 2012-04-20 CN CN201611072467.2A patent/CN107012171B/zh active Active
- 2012-04-20 DK DK21201945.9T patent/DK4005603T3/da active
- 2012-04-20 HU HUE20151479A patent/HUE057102T2/hu unknown
- 2012-04-20 HU HUE12774323A patent/HUE034510T2/en unknown
- 2012-04-20 SG SG10202110919YA patent/SG10202110919YA/en unknown
- 2012-04-20 FI FIEP21201945.9T patent/FI4005603T3/fi active
- 2012-04-20 HR HRP20171334TT patent/HRP20171334T1/hr unknown
- 2012-04-20 RS RS20241274A patent/RS66184B1/sr unknown
- 2012-04-20 EP EP17168534.0A patent/EP3254703B1/en active Active
- 2012-04-20 EP EP20151479.1A patent/EP3693025B1/en active Active
- 2012-04-20 RS RS20220014A patent/RS62795B1/sr unknown
- 2012-04-20 KR KR1020207002952A patent/KR102202908B1/ko active Active
- 2012-04-20 HU HUE21201945A patent/HUE069642T2/hu unknown
- 2012-04-20 DK DK12774323.5T patent/DK2699270T3/en active
- 2012-04-20 CA CA2833870A patent/CA2833870C/en active Active
- 2012-04-20 US US14/113,205 patent/US9193956B2/en active Active
- 2012-04-20 KR KR1020137030816A patent/KR102121355B1/ko active Active
- 2012-04-20 SG SG2013078597A patent/SG194583A1/en unknown
- 2012-04-20 EP EP21201945.9A patent/EP4005603B1/en active Active
- 2012-04-20 HU HUE17168534A patent/HUE049629T2/hu unknown
- 2012-04-20 BR BR112013027120-5A patent/BR112013027120B1/pt active IP Right Grant
- 2012-04-20 CA CA3069946A patent/CA3069946C/en active Active
- 2012-04-20 LT LTEP20151479.1T patent/LT3693025T/lt unknown
- 2012-04-20 ES ES21201945T patent/ES2994914T3/es active Active
- 2012-04-20 RU RU2013151885A patent/RU2611202C2/ru active
- 2012-04-20 DK DK17168534.0T patent/DK3254703T3/da active
- 2012-04-20 HR HRP20220036TT patent/HRP20220036T1/hr unknown
- 2012-04-20 KR KR1020227013781A patent/KR20220056884A/ko not_active Abandoned
- 2012-04-20 PT PT171685340T patent/PT3254703T/pt unknown
- 2012-04-20 KR KR1020217000600A patent/KR102391725B1/ko active Active
- 2012-04-20 PL PL17168534T patent/PL3254703T3/pl unknown
- 2012-04-20 EP EP12774323.5A patent/EP2699270B1/en active Active
- 2012-04-20 WO PCT/US2012/034413 patent/WO2012145601A2/en not_active Ceased
- 2012-04-20 SM SM20220036T patent/SMT202200036T1/it unknown
- 2012-04-20 PL PL12774323T patent/PL2699270T3/pl unknown
- 2012-04-20 EP EP24200481.0A patent/EP4471424A3/en active Pending
- 2012-04-20 LT LTEP21201945.9T patent/LT4005603T/lt unknown
- 2012-04-20 DK DK20151479.1T patent/DK3693025T3/da active
- 2012-04-20 RS RS20170855A patent/RS56246B1/sr unknown
- 2012-04-20 CN CN201610137255.1A patent/CN105755044A/zh active Pending
- 2012-04-20 SI SI201231053T patent/SI2699270T1/sl unknown
- 2012-04-20 RS RS20200498A patent/RS60207B1/sr unknown
-
2013
- 2013-10-20 IL IL228976A patent/IL228976B/en active IP Right Grant
- 2013-10-24 ZA ZA2013/07968A patent/ZA201307968B/en unknown
-
2014
- 2014-07-28 US US14/444,375 patent/US20140364338A1/en not_active Abandoned
- 2014-12-11 ZA ZA2014/09129A patent/ZA201409129B/en unknown
-
2015
- 2015-01-27 US US14/606,543 patent/US9856539B2/en active Active
- 2015-04-30 US US14/701,063 patent/US9458517B2/en active Active
- 2015-11-11 US US14/938,154 patent/US9587282B2/en active Active
-
2016
- 2016-08-23 US US15/244,884 patent/US10202657B2/en active Active
- 2016-08-23 US US15/244,892 patent/US10214785B2/en active Active
- 2016-12-13 AU AU2016273850A patent/AU2016273850B2/en active Active
- 2016-12-28 JP JP2016254766A patent/JP2017113002A/ja active Pending
-
2017
- 2017-02-08 RU RU2017104094A patent/RU2742724C9/ru active
- 2017-09-20 CY CY20171100993T patent/CY1119765T1/el unknown
-
2018
- 2018-09-25 AU AU2018236725A patent/AU2018236725B2/en active Active
- 2018-12-21 US US16/230,080 patent/US11236402B2/en active Active
-
2019
- 2019-12-02 JP JP2019217747A patent/JP2020028308A/ja not_active Withdrawn
-
2020
- 2020-05-11 HR HRP20200762TT patent/HRP20200762T1/hr unknown
- 2020-05-12 CY CY20201100437T patent/CY1122902T1/el unknown
- 2020-09-28 AU AU2020244399A patent/AU2020244399B2/en active Active
-
2021
- 2021-05-28 JP JP2021090666A patent/JP2021121638A/ja active Pending
- 2021-12-14 US US17/551,030 patent/US20220243291A1/en active Pending
-
2022
- 2022-01-12 CY CY20221100029T patent/CY1124905T1/el unknown
- 2022-07-05 JP JP2022108206A patent/JP2022125201A/ja active Pending
-
2024
- 2024-12-27 JP JP2024232069A patent/JP2025039615A/ja active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| ZA201409129B (en) | Adeno-associated virus virions with variant capsid and methods of use thereof | |
| IL262123A (en) | Influenza virus vaccines and their uses | |
| ZA201404797B (en) | Influenza virus vaccines and uses thereof | |
| EP2552479A4 (en) | INFLUENZA VIRUS VACCINES AND USES THEREOF | |
| IL236838B (en) | Antibodies against dengue virus and uses thereof | |
| IL239479A0 (en) | Influenza virus vaccines and their use | |
| SG11201404313YA (en) | Biomarkers and combination therapies using oncolytic virus and immunomodulation | |
| SG11201504805PA (en) | Porcine parvovirus 5a, methods of use and vaccine | |
| SG10201602184TA (en) | Treatment of hepatitis c virus infection with alisporivir | |
| ZA201306878B (en) | Treatment of hepatitis c virus infection with alisporivir | |
| TH1201001748B (th) | ส่วนสร้างที่สามารถผลิตแคปสิดเปล่าของไวรัส |