CA3216419A1 - Tissue-targeted modified aav capsids and methods of use thereof - Google Patents
Tissue-targeted modified aav capsids and methods of use thereof Download PDFInfo
- Publication number
- CA3216419A1 CA3216419A1 CA3216419A CA3216419A CA3216419A1 CA 3216419 A1 CA3216419 A1 CA 3216419A1 CA 3216419 A CA3216419 A CA 3216419A CA 3216419 A CA3216419 A CA 3216419A CA 3216419 A1 CA3216419 A1 CA 3216419A1
- Authority
- CA
- Canada
- Prior art keywords
- aav
- seq
- amino acid
- capsid protein
- protein
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
-
- 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
-
- 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
- C12N15/63—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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Virology (AREA)
- Zoology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Toxicology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163178965P | 2021-04-23 | 2021-04-23 | |
| US63/178,965 | 2021-04-23 | ||
| US202263299697P | 2022-01-14 | 2022-01-14 | |
| US63/299,697 | 2022-01-14 | ||
| PCT/US2022/026048 WO2022226374A1 (en) | 2021-04-23 | 2022-04-22 | Tissue-targeted modified aav capsids and methods of use thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3216419A1 true CA3216419A1 (en) | 2022-10-27 |
Family
ID=81598018
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3216419A Pending CA3216419A1 (en) | 2021-04-23 | 2022-04-22 | Tissue-targeted modified aav capsids and methods of use thereof |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20240197918A1 (https=) |
| EP (1) | EP4326868A1 (https=) |
| JP (1) | JP2024514956A (https=) |
| CA (1) | CA3216419A1 (https=) |
| WO (1) | WO2022226374A1 (https=) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20230159949A1 (en) * | 2020-07-22 | 2023-05-25 | The Broad Institute, Inc. | Engineered muscle targeting compositions |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| EP4353737A1 (en) * | 2022-10-10 | 2024-04-17 | Universitätsklinikum Hamburg-Eppendorf | Fusion proteins comprising a cell surface marker specific vhh |
| TW202502803A (zh) | 2023-03-10 | 2025-01-16 | 美商戴諾治療公司 | 衣殼多肽及其使用方法 |
| CN118955652A (zh) * | 2023-05-15 | 2024-11-15 | 杭州新昶基因技术有限公司 | 腺相关病毒衣壳蛋白、包含其的腺相关病毒及应用 |
| WO2024238807A2 (en) * | 2023-05-16 | 2024-11-21 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| TW202525832A (zh) | 2023-08-31 | 2025-07-01 | 美商戴諾治療公司 | 衣殼多肽及其使用方法(二) |
| WO2025137378A1 (en) * | 2023-12-20 | 2025-06-26 | Aavantibio, Inc. | Aav capsid engineering for cardiac and/or musculoskeletal gene therapy |
| WO2025133247A1 (en) * | 2023-12-21 | 2025-06-26 | Genethon | Methods for aav vector re-administration |
| TW202545971A (zh) | 2024-02-08 | 2025-12-01 | 美商戴諾治療公司 | 殼體多肽及其使用方法 |
| WO2025185625A1 (zh) * | 2024-03-04 | 2025-09-12 | 苏州新芽基因生物技术有限公司 | 特异性靶向人组织的多肽序列及其在构建递送载体中的应用 |
| WO2025230000A1 (ja) * | 2024-05-01 | 2025-11-06 | Ｊｃｒファーマ株式会社 | 修飾されたａａｖ粒子、その製造方法、関連する医薬及びその製造 |
| WO2026064444A1 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2026064428A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2026064442A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2026069251A1 (en) * | 2024-09-30 | 2026-04-02 | Sarepta Therapeutics, Inc. | Adeno-associated virus capsid antibodies and fragments thereof |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| CN101895633A (zh) | 2010-07-14 | 2010-11-24 | 中兴通讯股份有限公司 | 一种移动终端及其解锁方法 |
| WO2012068627A1 (en) | 2010-11-24 | 2012-05-31 | The University Of Western Australia | Peptides for the specific binding of rna targets |
| 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 |
| WO2013058404A1 (ja) | 2011-10-21 | 2013-04-25 | 国立大学法人九州大学 | Pprモチーフを利用したrna結合性蛋白質の設計方法及びその利用 |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| US10330674B2 (en) | 2015-01-13 | 2019-06-25 | Massachusetts Institute Of Technology | Pumilio domain-based modular protein architecture for RNA binding |
| AU2020368539A1 (en) * | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| AU2021264061A1 (en) * | 2020-05-01 | 2022-12-01 | Massachusetts Institute Of Technology | Engineered central nervous system compositions |
| TW202217003A (zh) * | 2020-07-22 | 2022-05-01 | 美商博得學院股份有限公司 | 經改造之肌肉靶向組合物 |
-
2022
- 2022-04-22 CA CA3216419A patent/CA3216419A1/en active Pending
- 2022-04-22 EP EP22722646.1A patent/EP4326868A1/en active Pending
- 2022-04-22 WO PCT/US2022/026048 patent/WO2022226374A1/en not_active Ceased
- 2022-04-22 JP JP2023565273A patent/JP2024514956A/ja active Pending
- 2022-04-22 US US18/287,963 patent/US20240197918A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2024514956A (ja) | 2024-04-03 |
| WO2022226374A1 (en) | 2022-10-27 |
| EP4326868A1 (en) | 2024-02-28 |
| US20240197918A1 (en) | 2024-06-20 |
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