CA3193128A1 - Peptide-modified aav capsid - Google Patents
Peptide-modified aav capsidInfo
- Publication number
- CA3193128A1 CA3193128A1 CA3193128A CA3193128A CA3193128A1 CA 3193128 A1 CA3193128 A1 CA 3193128A1 CA 3193128 A CA3193128 A CA 3193128A CA 3193128 A CA3193128 A CA 3193128A CA 3193128 A1 CA3193128 A1 CA 3193128A1
- Authority
- CA
- Canada
- Prior art keywords
- gene
- sequence
- aav
- muscle
- aav9
- 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
- C07K14/005—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
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicinal Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP20306005.8 | 2020-09-10 | ||
| EP20306005 | 2020-09-10 | ||
| PCT/EP2021/074964 WO2022053630A1 (en) | 2020-09-10 | 2021-09-10 | Peptide-modified aav capsid |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3193128A1 true CA3193128A1 (en) | 2022-03-17 |
Family
ID=72717827
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3193128A Pending CA3193128A1 (en) | 2020-09-10 | 2021-09-10 | Peptide-modified aav capsid |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20240209030A1 (enExample) |
| EP (2) | EP4211231B1 (enExample) |
| JP (1) | JP7834725B2 (enExample) |
| CN (1) | CN116096431A (enExample) |
| CA (1) | CA3193128A1 (enExample) |
| DK (1) | DK4211231T3 (enExample) |
| ES (1) | ES2984259T3 (enExample) |
| FI (1) | FI4211231T3 (enExample) |
| PL (1) | PL4211231T3 (enExample) |
| PT (1) | PT4211231T (enExample) |
| WO (1) | WO2022053630A1 (enExample) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2021259422A1 (en) | 2020-04-20 | 2022-12-08 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| WO2022204476A1 (en) | 2021-03-26 | 2022-09-29 | The Board Of Regents Of The University Of Texas System | Nucleotide editing to reframe dmd transcripts by base editing and prime editing |
| WO2023012313A1 (en) | 2021-08-04 | 2023-02-09 | Genethon | Hybrid promoters for gene expression in muscles and in the cns |
| EP4504952A4 (en) * | 2022-04-07 | 2026-04-08 | Kate Therapeutics Inc | COMPOSITIONS AND METHODS FOR MUSCULAR DISORDERS |
| 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 |
| WO2023237731A1 (en) | 2022-06-09 | 2023-12-14 | Genethon | N-terminal truncated gde for the treatment of glycogen storage disease iii |
| FI4536686T3 (fi) | 2022-06-10 | 2026-01-13 | Genethon | Lihaksen parannetun transduktiotehokkuuden omaava peptidimodifioitu aav-kapsidi |
| JP2025532754A (ja) | 2022-10-07 | 2025-10-03 | ジェネトン | 抗ｆａｐ ｃａｒ－ｔ細胞を使用する骨格筋ミオパチーの免疫療法 |
| JPWO2024080311A1 (enExample) * | 2022-10-12 | 2024-04-18 | ||
| JP2026509271A (ja) | 2023-03-10 | 2026-03-17 | ダイノ セラピューティクス，インコーポレーテッド | カプシドポリペプチドおよびその使用方法 |
| WO2024188913A1 (en) | 2023-03-10 | 2024-09-19 | Genethon | Induction of immune tolerance by aav vector comprising the combination of a liver detargeted capsid and a tandem liver-muscle specific promoter |
| WO2025003477A1 (en) | 2023-06-29 | 2025-01-02 | Genethon | N-terminal truncated glycogen debranching enzymes for the treatment of glycogen storage disease iii |
| EP4766844A1 (en) * | 2023-08-25 | 2026-07-01 | Agency for Science, Technology And Research | COMPOSITION AND METHODS FOR IDENTIFYING NEW AAV VARIANTS FOR ADMINISTRATING DIFFERENTIATED GENES TO CARDIOMYOCTS AND/OR SKELETAL MYOFIBERS |
| WO2025133247A1 (en) | 2023-12-21 | 2025-06-26 | Genethon | Methods for aav vector re-administration |
| KR102933125B1 (ko) * | 2024-02-01 | 2026-03-04 | 주식회사 글루진테라퓨틱스 | 평활근 세포 특이적 유전자 전달이 가능한 아데노연관바이러스 벡터 |
| 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 |
| WO2026017727A1 (en) | 2024-07-16 | 2026-01-22 | Genethon | Alternative barcoding method to assess transduction efficiency of aav vectors |
| CN119751596B (zh) * | 2024-07-16 | 2025-08-01 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2026064428A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2026064444A1 (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 |
| WO2026122963A1 (en) | 2024-12-06 | 2026-06-11 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015006743A1 (en) * | 2013-07-12 | 2015-01-15 | The Children's Hospital Of Philadelphia | Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies |
| AU2016366549B2 (en) * | 2015-12-11 | 2022-11-10 | California Institute Of Technology | Targeting peptides for directing adeno-associated viruses (AAVs) |
| JP6994018B2 (ja) * | 2016-07-26 | 2022-01-14 | バイオマリン ファーマシューティカル インコーポレイテッド | 新規アデノ随伴ウイルスキャプシドタンパク質 |
| JP7374119B2 (ja) * | 2018-04-05 | 2023-11-06 | ジェネトン | 減少した肝臓向性を有するＡＡＶ９とＡＡＶｒｈ７４とのハイブリッド組換えアデノ随伴ウイルス血清型 |
| CA3097375A1 (en) | 2018-04-27 | 2019-10-31 | Universitat Heidelberg | Modified aav capsid polypeptides for treatment of muscular diseases |
-
2021
- 2021-09-10 CN CN202180055064.XA patent/CN116096431A/zh active Pending
- 2021-09-10 CA CA3193128A patent/CA3193128A1/en active Pending
- 2021-09-10 FI FIEP21777271.4T patent/FI4211231T3/fi active
- 2021-09-10 ES ES21777271T patent/ES2984259T3/es active Active
- 2021-09-10 PT PT217772714T patent/PT4211231T/pt unknown
- 2021-09-10 PL PL21777271.4T patent/PL4211231T3/pl unknown
- 2021-09-10 EP EP21777271.4A patent/EP4211231B1/en active Active
- 2021-09-10 WO PCT/EP2021/074964 patent/WO2022053630A1/en not_active Ceased
- 2021-09-10 EP EP24183857.2A patent/EP4421179A3/en not_active Withdrawn
- 2021-09-10 US US18/024,103 patent/US20240209030A1/en active Pending
- 2021-09-10 JP JP2023515813A patent/JP7834725B2/ja active Active
- 2021-09-10 DK DK21777271.4T patent/DK4211231T3/da active
Also Published As
| Publication number | Publication date |
|---|---|
| FI4211231T3 (fi) | 2024-07-17 |
| CN116096431A (zh) | 2023-05-09 |
| ES2984259T3 (es) | 2024-10-29 |
| US20240209030A1 (en) | 2024-06-27 |
| EP4211231A1 (en) | 2023-07-19 |
| JP2023544683A (ja) | 2023-10-25 |
| WO2022053630A1 (en) | 2022-03-17 |
| DK4211231T3 (da) | 2024-07-08 |
| EP4421179A3 (en) | 2024-12-18 |
| JP7834725B2 (ja) | 2026-03-24 |
| EP4421179A2 (en) | 2024-08-28 |
| BR112023004176A2 (pt) | 2023-04-11 |
| PL4211231T3 (pl) | 2024-11-04 |
| EP4211231B1 (en) | 2024-06-26 |
| PT4211231T (pt) | 2024-07-24 |
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Legal Events
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