IL301563A - Recombinant adeno-associated viruses for cns or muscle delivery - Google Patents
Recombinant adeno-associated viruses for cns or muscle deliveryInfo
- Publication number
- IL301563A IL301563A IL301563A IL30156323A IL301563A IL 301563 A IL301563 A IL 301563A IL 301563 A IL301563 A IL 301563A IL 30156323 A IL30156323 A IL 30156323A IL 301563 A IL301563 A IL 301563A
- Authority
- IL
- Israel
- Prior art keywords
- capsid
- aav
- seq
- aav9
- raav
- Prior art date
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- 108091005703 transmembrane proteins Proteins 0.000 description 1
- 230000009529 traumatic brain injury Effects 0.000 description 1
- 229960004791 tropicamide Drugs 0.000 description 1
- 230000006107 tyrosine sulfation Effects 0.000 description 1
- 241000701447 unidentified baculovirus Species 0.000 description 1
- 238000011144 upstream manufacturing Methods 0.000 description 1
- 210000001186 vagus nerve Anatomy 0.000 description 1
- 210000003556 vascular endothelial cell Anatomy 0.000 description 1
- 210000005166 vasculature Anatomy 0.000 description 1
- 235000013311 vegetables Nutrition 0.000 description 1
- 210000002845 virion Anatomy 0.000 description 1
- 238000012800 visualization Methods 0.000 description 1
- 230000003442 weekly effect Effects 0.000 description 1
- 239000000080 wetting agent Substances 0.000 description 1
Classifications
-
- 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
-
- 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
- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- 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
- 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
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurosurgery (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063088988P | 2020-10-07 | 2020-10-07 | |
| PCT/US2021/054058 WO2022076750A2 (en) | 2020-10-07 | 2021-10-07 | Recombinant adeno-associated viruses for cns or muscle delivery |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL301563A true IL301563A (en) | 2023-05-01 |
Family
ID=86184347
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL301563A IL301563A (en) | 2020-10-07 | 2021-10-07 | Recombinant adeno-associated viruses for cns or muscle delivery |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230374541A1 (https=) |
| EP (1) | EP4225778A2 (https=) |
| JP (1) | JP2023545384A (https=) |
| KR (1) | KR20230082613A (https=) |
| CN (1) | CN116783209A (https=) |
| AU (1) | AU2021358546A1 (https=) |
| CA (1) | CA3193685A1 (https=) |
| IL (1) | IL301563A (https=) |
| MX (1) | MX2023003807A (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20260055144A1 (en) * | 2022-08-24 | 2026-02-26 | Regenxbio Inc. | Recombinant adeno-associated viruses and uses thereof |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018119330A2 (en) * | 2016-12-22 | 2018-06-28 | Oregon Health & Science University | Adeno associated viral vectors |
| US11905312B2 (en) * | 2017-02-15 | 2024-02-20 | The University Of North Carolina At Chapel Hill | Methods and compositions for gene transfer across the vasculature |
| WO2020160508A1 (en) * | 2019-01-31 | 2020-08-06 | Oregon Health & Science University | Methods for using transcription-dependent directed evolution of aav capsids |
-
2021
- 2021-10-07 KR KR1020237010841A patent/KR20230082613A/ko active Pending
- 2021-10-07 JP JP2023519433A patent/JP2023545384A/ja active Pending
- 2021-10-07 IL IL301563A patent/IL301563A/en unknown
- 2021-10-07 MX MX2023003807A patent/MX2023003807A/es unknown
- 2021-10-07 US US18/030,683 patent/US20230374541A1/en active Pending
- 2021-10-07 CN CN202180066787.XA patent/CN116783209A/zh active Pending
- 2021-10-07 AU AU2021358546A patent/AU2021358546A1/en not_active Abandoned
- 2021-10-07 EP EP21802102.0A patent/EP4225778A2/en active Pending
- 2021-10-07 CA CA3193685A patent/CA3193685A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| MX2023003807A (es) | 2023-04-12 |
| EP4225778A2 (en) | 2023-08-16 |
| CA3193685A1 (en) | 2022-04-14 |
| AU2021358546A9 (en) | 2024-02-08 |
| US20230374541A1 (en) | 2023-11-23 |
| CN116783209A (zh) | 2023-09-19 |
| AU2021358546A1 (en) | 2023-06-08 |
| KR20230082613A (ko) | 2023-06-08 |
| JP2023545384A (ja) | 2023-10-30 |
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