AU2017341849B2 - AAV capsid designs - Google Patents
AAV capsid designs Download PDFInfo
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- AU2017341849B2 AU2017341849B2 AU2017341849A AU2017341849A AU2017341849B2 AU 2017341849 B2 AU2017341849 B2 AU 2017341849B2 AU 2017341849 A AU2017341849 A AU 2017341849A AU 2017341849 A AU2017341849 A AU 2017341849A AU 2017341849 B2 AU2017341849 B2 AU 2017341849B2
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- hsa
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- aav
- raav
- transgene
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- 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
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
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- A—HUMAN NECESSITIES
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- 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
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- 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
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- 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
- C07K14/01—DNA viruses
- C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
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- 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
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
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- 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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
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- 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
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
- C12N2015/8518—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic expressing industrially exogenous proteins, e.g. for pharmaceutical use, human insulin, blood factors, immunoglobulins, pseudoparticles
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- 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
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14123—Virus like particles [VLP]
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Virology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
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- Biophysics (AREA)
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- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Diabetes (AREA)
- Neurology (AREA)
- Cardiology (AREA)
- Hematology (AREA)
- Urology & Nephrology (AREA)
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2024203491A AU2024203491A1 (en) | 2016-10-13 | 2024-05-24 | AAV capsid designs |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662408022P | 2016-10-13 | 2016-10-13 | |
| US62/408,022 | 2016-10-13 | ||
| US201662417756P | 2016-11-04 | 2016-11-04 | |
| US62/417,756 | 2016-11-04 | ||
| US201762486642P | 2017-04-18 | 2017-04-18 | |
| US62/486,642 | 2017-04-18 | ||
| PCT/US2017/056614 WO2018071831A1 (en) | 2016-10-13 | 2017-10-13 | Aav capsid designs |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2024203491A Division AU2024203491A1 (en) | 2016-10-13 | 2024-05-24 | AAV capsid designs |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2017341849A1 AU2017341849A1 (en) | 2019-05-02 |
| AU2017341849B2 true AU2017341849B2 (en) | 2024-03-21 |
Family
ID=61906092
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2017341849A Active AU2017341849B2 (en) | 2016-10-13 | 2017-10-13 | AAV capsid designs |
| AU2024203491A Pending AU2024203491A1 (en) | 2016-10-13 | 2024-05-24 | AAV capsid designs |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2024203491A Pending AU2024203491A1 (en) | 2016-10-13 | 2024-05-24 | AAV capsid designs |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US11578340B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3526333A4 (cg-RX-API-DMAC7.html) |
| JP (2) | JP7577291B6 (cg-RX-API-DMAC7.html) |
| KR (2) | KR20190075964A (cg-RX-API-DMAC7.html) |
| CN (1) | CN110506119A (cg-RX-API-DMAC7.html) |
| AU (2) | AU2017341849B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3040483A1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2018071831A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| RU2738421C2 (ru) | 2014-10-21 | 2020-12-14 | Юниверсити Оф Массачусетс | Варианты рекомбинантных aav и их применения |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| US20200316221A1 (en) * | 2016-10-13 | 2020-10-08 | University Of Massachusetts | Aav capsid designs |
| WO2018071831A1 (en) | 2016-10-13 | 2018-04-19 | University Of Massachusetts | Aav capsid designs |
| US20230048025A1 (en) * | 2016-12-22 | 2023-02-16 | Oregon Health & Science University | Adeno associated viral vectors |
| CN110462037A (zh) * | 2017-01-30 | 2019-11-15 | 学校法人日本医科大学 | 腺相关病毒(aav)衣壳蛋白的突变体 |
| MX2019012113A (es) | 2017-04-14 | 2020-09-10 | Univ Nat Taiwan | Terapia génica para la deficiencia de la aadc. |
| SG11202009854XA (en) * | 2018-02-15 | 2020-11-27 | Bjoerklund Tomas | Modified viral capsids |
| EP3758725A4 (en) | 2018-02-28 | 2021-05-19 | University of Pittsburgh - of the Commonwealth System of Higher Education | MODULAR SYSTEM FOR GENE AND PROTEIN ADMINISTRATION BASED ON AAV |
| ES3050313T3 (en) * | 2018-05-11 | 2025-12-19 | Massachusetts Eye & Ear Infirmary | Liver-specific tropism of adeno-associated viruses |
| CN113966399A (zh) | 2018-09-26 | 2022-01-21 | 加州理工学院 | 用于靶向基因疗法的腺相关病毒组合物 |
| US20220073905A1 (en) * | 2018-12-28 | 2022-03-10 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Methods and materials for single cell transcriptome-based development of aav vectors and promoters |
| WO2020223276A1 (en) * | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of tauopathy |
| AU2020299026A1 (en) * | 2019-07-04 | 2022-01-20 | Children's Medical Research Institute | Methods and AAV vectors for in vivo transduction |
| KR102167829B1 (ko) * | 2020-02-10 | 2020-10-20 | 주식회사 이노테라피 | 아데노연관바이러스용 안정화제 및 이를 이용한 아데노연관바이러스의 안정화 방법 |
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