CA3182970A1 - Redirection of tropism of aav capsids - Google Patents
Redirection of tropism of aav capsidsInfo
- Publication number
- CA3182970A1 CA3182970A1 CA3182970A CA3182970A CA3182970A1 CA 3182970 A1 CA3182970 A1 CA 3182970A1 CA 3182970 A CA3182970 A CA 3182970A CA 3182970 A CA3182970 A CA 3182970A CA 3182970 A1 CA3182970 A1 CA 3182970A1
- Authority
- CA
- Canada
- Prior art keywords
- seq
- amino acid
- acid sequence
- sequence
- aav capsid
- 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
-
- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- C—CHEMISTRY; METALLURGY
- C40—COMBINATORIAL TECHNOLOGY
- C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
- C40B40/00—Libraries per se, e.g. arrays, mixtures
- C40B40/04—Libraries containing only organic compounds
- C40B40/06—Libraries containing nucleotides or polynucleotides, or derivatives thereof
- C40B40/08—Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
-
- 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
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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)
- Molecular Biology (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Mycology (AREA)
- Immunology (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063023927P | 2020-05-13 | 2020-05-13 | |
| US63/023,927 | 2020-05-13 | ||
| US202063122300P | 2020-12-07 | 2020-12-07 | |
| US63/122,300 | 2020-12-07 | ||
| PCT/US2021/025061 WO2021230987A1 (en) | 2020-05-13 | 2021-03-31 | Redirection of tropism of aav capsids |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3182970A1 true CA3182970A1 (en) | 2021-11-18 |
Family
ID=75639982
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3182970A Pending CA3182970A1 (en) | 2020-05-13 | 2021-03-31 | Redirection of tropism of aav capsids |
Country Status (14)
| Country | Link |
|---|---|
| US (3) | US20230203102A1 (enExample) |
| EP (1) | EP4149955A1 (enExample) |
| JP (2) | JP7731909B2 (enExample) |
| KR (1) | KR20230022175A (enExample) |
| CN (1) | CN116096734A (enExample) |
| AU (1) | AU2021273447B2 (enExample) |
| BR (1) | BR112022023106A2 (enExample) |
| CA (1) | CA3182970A1 (enExample) |
| CO (1) | CO2022016156A2 (enExample) |
| IL (1) | IL298001A (enExample) |
| MX (1) | MX2022014256A (enExample) |
| PE (1) | PE20230767A1 (enExample) |
| TW (1) | TWI900553B (enExample) |
| WO (1) | WO2021230987A1 (enExample) |
Families Citing this family (80)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2525067T3 (es) * | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| CN113166208B (zh) | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| JP2022513067A (ja) * | 2018-11-16 | 2022-02-07 | アスクレピオス バイオファーマシューティカル， インコーポレイテッド | ポンペ病を処置するための治療用アデノ随伴ウイルス |
| TWI900553B (zh) | 2020-05-13 | 2025-10-11 | 美商航海家醫療公司 | Aav蛋白殼之趨性重定向 |
| US20230295238A1 (en) * | 2020-07-23 | 2023-09-21 | The Scripps Research Institute | Insulin receptor-mediated enhancement of gene transfer |
| EP4291654A2 (en) | 2021-02-12 | 2023-12-20 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
| WO2022187473A2 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| WO2022187548A1 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| JP2024533174A (ja) * | 2021-09-03 | 2024-09-12 | ビオマリン プハルマセウトイカル インコーポレイテッド | Ａａｖカプシド組成物及び送達方法 |
| WO2023044483A2 (en) | 2021-09-20 | 2023-03-23 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| KR20240095539A (ko) | 2021-10-08 | 2024-06-25 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 변이체 및 이의 사용 방법 |
| EP4426716A1 (en) | 2021-11-02 | 2024-09-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| US20250049955A1 (en) | 2021-11-17 | 2025-02-13 | Voyager Therapeutics, Inc. | Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| WO2023092002A2 (en) * | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis and disorders associatedwith the spinal cord |
| EP4433169A1 (en) | 2021-11-17 | 2024-09-25 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| US20250034559A1 (en) | 2021-11-17 | 2025-01-30 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of tau-related disorders |
| WO2023147374A2 (en) | 2022-01-25 | 2023-08-03 | Voyager Therapeutics, Inc. | Baculovirus expression system |
| US20250297280A2 (en) * | 2022-02-01 | 2025-09-25 | The Broad Institute, Inc. | Adeno-associated viral vectors and uses thereof |
| CA3249551A1 (en) | 2022-02-08 | 2023-08-17 | Voyager Therapeutics, Inc. | AAV Capsid Variants and Their Uses |
| EP4504754A2 (en) * | 2022-04-04 | 2025-02-12 | Vectory B.V. | Recombinant aav capsid proteins |
| WO2023220695A2 (en) | 2022-05-13 | 2023-11-16 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| EP4522633A2 (en) * | 2022-05-13 | 2025-03-19 | The Broad Institute, Inc. | Adeno-associated viral vectors and uses thereof |
| EP4526324A1 (en) * | 2022-05-18 | 2025-03-26 | The Broad Institute, Inc. | Engineered viral capsids with increased stability and methods of use thereof |
| EP4532515A1 (en) | 2022-06-02 | 2025-04-09 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
| EP4543473A1 (en) | 2022-06-22 | 2025-04-30 | Voyager Therapeutics, Inc. | Tau binding compounds |
| CA3259902A1 (en) | 2022-06-28 | 2024-01-04 | Voyager Therapeutics, Inc. | AAV Capsid Variants and Their Uses |
| US20250388626A1 (en) | 2022-07-06 | 2025-12-25 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| JP2025528068A (ja) | 2022-08-03 | 2025-08-26 | ボイジャー セラピューティクス インコーポレイテッド | 血液脳関門を通過させるための組成物及び方法 |
| US20260085328A1 (en) | 2022-09-08 | 2026-03-26 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| WO2024055020A2 (en) * | 2022-09-09 | 2024-03-14 | The Regents Of The University Of California | Reprogramming tropism via displayed peptides tiling receptor-ligands |
| KR20250069606A (ko) | 2022-09-15 | 2025-05-19 | 보이저 테라퓨틱스, 인크. | 타우 결합 화합물 |
| WO2024086747A1 (en) | 2022-10-19 | 2024-04-25 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| AU2023378899A1 (en) | 2022-11-13 | 2025-06-26 | Alexion Pharma International Operations Limited | Gene therapy for frontotemporal dementia |
| CN118420721A (zh) * | 2023-01-31 | 2024-08-02 | 上海朗昇生物科技有限公司 | 一种组织嗜性的腺相关病毒衣壳及其用途 |
| AU2023427408A1 (en) | 2023-02-02 | 2025-09-04 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| WO2024191778A1 (en) | 2023-03-10 | 2024-09-19 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2024199270A1 (zh) * | 2023-03-27 | 2024-10-03 | 浙江大学 | 氨基酸序列的生成与筛选方法及其系统 |
| WO2024226761A2 (en) | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| WO2024226790A1 (en) | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| AU2024266140A1 (en) | 2023-05-02 | 2025-09-11 | Voyager Therapeutics, Inc. | Compositions and methods for regulating mapt |
| WO2024229173A2 (en) | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to ataxin-2 |
| EP4705484A1 (en) | 2023-05-03 | 2026-03-11 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of tau-related disorders |
| AU2024266519A1 (en) | 2023-05-03 | 2025-12-04 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency |
| KR20260021096A (ko) | 2023-05-03 | 2026-02-12 | 보이저 테라퓨틱스, 인크. | Cdkl5 결핍과 관련된 장애의 치료를 위한 조성물 및 방법 |
| EP4705487A2 (en) | 2023-05-03 | 2026-03-11 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to frataxin deficiency |
| WO2024229164A2 (en) | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| 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 |
| WO2025038795A1 (en) | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025038796A1 (en) | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to cdkl5 deficiency |
| TW202523846A (zh) | 2023-08-16 | 2025-06-16 | 美商航海家醫療公司 | 用於治療與共濟蛋白(frataxin)不足有關之疾病之組合物及方法 |
| WO2025038430A1 (en) | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2025038802A1 (en) | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to ataxin-2 |
| WO2025038805A1 (en) | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency |
| AU2024332181A1 (en) | 2023-08-31 | 2026-02-12 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| CN119569833A (zh) * | 2023-09-06 | 2025-03-07 | 北京锦篮基因科技有限公司 | 靶向骨骼肌的aav衣壳蛋白变体及其用途 |
| CN117264912A (zh) * | 2023-09-25 | 2023-12-22 | 中国科学院深圳先进技术研究院 | 一种重组腺相关病毒颗粒、重组腺相关病毒载体系统及其应用 |
| CN121925426A (zh) * | 2023-09-26 | 2026-04-24 | 凯特治疗学股份有限公司 | 肝脏去靶向衣壳 |
| WO2025072698A2 (en) * | 2023-09-27 | 2025-04-03 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Aav9 vectors and capsid polypeptides for use in treating cns disorders |
| WO2025122644A1 (en) | 2023-12-05 | 2025-06-12 | Voyager Therapeutics, Inc. | Compositions and methods for regulating mapt |
| WO2025122634A1 (en) | 2023-12-05 | 2025-06-12 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of tau-related disorders |
| WO2025122548A1 (en) | 2023-12-05 | 2025-06-12 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to cdkl5 deficiency |
| WO2025122543A1 (en) | 2023-12-05 | 2025-06-12 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency |
| WO2025122536A1 (en) | 2023-12-05 | 2025-06-12 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025122530A1 (en) | 2023-12-05 | 2025-06-12 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency |
| WO2025122532A1 (en) | 2023-12-05 | 2025-06-12 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to ataxin-2 |
| TW202540436A (zh) | 2023-12-05 | 2025-10-16 | 美商航海家醫療公司 | 用於治療與共濟蛋白(frataxin)缺陷相關之病症之組合物及方法 |
| WO2025137219A1 (en) | 2023-12-21 | 2025-06-26 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025147436A1 (en) | 2024-01-03 | 2025-07-10 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| 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 |
| WO2025194082A1 (en) * | 2024-03-15 | 2025-09-18 | The Children's Medical Center Corporation | Compositions and methods for delivering nucleic acids to podocytes |
| WO2025207954A1 (en) * | 2024-03-27 | 2025-10-02 | General Medicines Llc | Compositions and methods for improved adeno-associated viral gene therapy |
| CN118324853B (zh) * | 2024-04-03 | 2025-03-14 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2025214359A1 (en) * | 2024-04-09 | 2025-10-16 | Hangzhou Jiayin Biotech Ltd. | A novel aav variant |
| WO2025235734A2 (en) | 2024-05-09 | 2025-11-13 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2026012095A1 (en) * | 2024-06-21 | 2026-01-15 | Westlake Genetech. Ltd. | Adeno-associated virus variant capsids |
| WO2026010722A1 (en) | 2024-07-03 | 2026-01-08 | Voyager Therapeutics, Inc. | Compositions and methods for regulating mapt |
| WO2026050402A1 (en) * | 2024-08-29 | 2026-03-05 | Kate Therapeutics, Inc. | Liver de-targeted muscle tropic capsids |
| WO2026064442A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
Family Cites Families (204)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2640638B1 (fr) | 1988-12-20 | 1991-02-15 | Commissariat Energie Atomique | Bioreacteur et dispositif pour la culture de cellules animales |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5625048A (en) | 1994-11-10 | 1997-04-29 | The Regents Of The University Of California | Modified green fluorescent proteins |
| US6924128B2 (en) | 1994-12-06 | 2005-08-02 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
| US5741657A (en) | 1995-03-20 | 1998-04-21 | The Regents Of The University Of California | Fluorogenic substrates for β-lactamase and methods of use |
| US6281010B1 (en) | 1995-06-05 | 2001-08-28 | The Trustees Of The University Of Pennsylvania | Adenovirus gene therapy vehicle and cell line |
| US5756283A (en) | 1995-06-05 | 1998-05-26 | The Trustees Of The University Of Pennsylvania | Method for improved production of recombinant adeno-associated viruses for gene therapy |
| US6506379B1 (en) | 1995-06-07 | 2003-01-14 | Ariad Gene Therapeutics, Inc. | Intramuscular delivery of recombinant AAV |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| 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 |
| WO1998022588A2 (en) | 1996-11-20 | 1998-05-28 | Introgen Therapeutics, Inc. | An improved method for the production and purification of adenoviral vectors |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| WO1999014354A1 (en) | 1997-09-19 | 1999-03-25 | The Trustees Of The University Of The Pennsylvania | Methods and vector constructs useful for production of recombinant aav |
| EP1015619A1 (en) | 1997-09-19 | 2000-07-05 | The Trustees Of The University Of Pennsylvania | Methods and cell line useful for production of recombinant adeno-associated viruses |
| EP1064393B1 (en) | 1998-03-20 | 2004-12-29 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US6953690B1 (en) | 1998-03-20 | 2005-10-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US6984517B1 (en) | 1998-05-28 | 2006-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector and uses thereof |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| FR2786478B1 (fr) | 1998-11-30 | 2000-12-22 | Commissariat Energie Atomique | Confinement du cesium et/ou du rubidium dans des ceramiques apatitiques |
| JP4693244B2 (ja) | 1999-03-18 | 2011-06-01 | 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 | 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法 |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| CA2375098A1 (en) | 1999-06-02 | 2000-12-14 | Trustees Of The University Of Pennsylvania | Compositions and methods useful for production of recombinant viruses which require helper viruses |
| US6365394B1 (en) | 1999-09-29 | 2002-04-02 | The Trustees Of The University Of Pennsylvania | Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus |
| JP2003523320A (ja) | 1999-09-29 | 2003-08-05 | 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 | ウイルスベクターの迅速なｐｅｇ改変方法、高められた遺伝子形質導入のための組成物、高められた物理的安定性を伴う組成物、およびそのための用途 |
| EP1373527A1 (en) | 2001-04-06 | 2004-01-02 | CropDesign N.V. | The use of double and opposite recombination sites for the single step cloning of two dna segments |
| EP1390490B1 (en) | 2001-05-24 | 2009-04-15 | Genzyme Corporation | Muscle-specific expression vectors |
| DK2573170T3 (en) | 2001-12-17 | 2018-04-09 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use |
| US6845866B2 (en) | 2002-10-10 | 2005-01-25 | N. Henning Zieger | Dispenser |
| US7605249B2 (en) | 2002-11-26 | 2009-10-20 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| CN101410519B (zh) | 2003-04-25 | 2013-04-24 | 免疫医疗有限责任公司 | 间质肺病毒株及其在疫苗制剂中以及用作抗原性序列表达载体的用途和繁殖病毒的方法 |
| US8927269B2 (en) | 2003-05-19 | 2015-01-06 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Avian adenoassociated virus and uses thereof |
| US7261544B2 (en) | 2003-05-21 | 2007-08-28 | Genzyme Corporation | Methods for producing preparations of recombinant AAV virions substantially free of empty capsids |
| SI1633772T1 (sl) | 2003-06-19 | 2016-06-30 | Genzyme Corporation | AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen |
| CA2528511C (en) | 2003-06-20 | 2015-11-03 | The Trustees Of The University Of Pennsylvania | Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses |
| US7291498B2 (en) | 2003-06-20 | 2007-11-06 | The Trustees Of The University Of Pennsylvania | Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses |
| 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 |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| US8137960B2 (en) | 2003-12-04 | 2012-03-20 | The United States Of America As Represented By The Department Of Health And Human Services | Bovine adeno-associated viral (BAAV) vector and uses thereof |
| US20060166363A1 (en) | 2004-01-27 | 2006-07-27 | Sergei Zolotukhin | Modified baculovirus expression system for production of pseudotyped rAAV vector |
| WO2005116204A1 (ja) | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法 |
| US7427396B2 (en) | 2004-06-03 | 2008-09-23 | Genzyme Corporation | AAV vectors for gene delivery to the lung |
| EP2311966A3 (en) | 2005-10-20 | 2012-09-05 | Amsterdam Molecular Therapeutics (AMT) B.V. | Improved AAV vectors produced in insect cells |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| WO2008024998A2 (en) | 2006-08-24 | 2008-02-28 | Virovek, Inc. | Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor |
| ES2602610T3 (es) | 2007-05-31 | 2017-02-21 | Medigene Ag | Proteína estructural mutada de un parvovirus |
| EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| EP2312608B1 (en) | 2008-07-09 | 2015-01-14 | Kabushiki Kaisha Toshiba | Target for x-ray tube, x-ray tube using the same, x-ray inspection system, and method for producing target for x-ray tube |
| DE102008060558B4 (de) | 2008-12-04 | 2013-09-26 | Eduard Buzetzki | Verfahren zum katalytischen Cracken von Pflanzenölen und tierischen Fetten |
| WO2010093784A2 (en) | 2009-02-11 | 2010-08-19 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| US9163261B2 (en) | 2010-02-22 | 2015-10-20 | Koteswara Rao KOLLIPARA | Adeno-associated virus 2/8—micro RNA-101 therapy for liver cancer |
| EP2547781A4 (en) | 2010-03-17 | 2014-04-23 | Anaptysbio Inc | PROCESS FOR PRODUCING TRANSCRIPTION PRODUCTS USING CRYPTIC SPLICE SITES |
| EP2561073B1 (en) | 2010-04-23 | 2016-08-24 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| US10415056B2 (en) | 2010-11-10 | 2019-09-17 | Fred Hutchinson Cancer Research Center | Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination |
| 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 |
| CN105755044A (zh) | 2011-04-22 | 2016-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| ES2857773T5 (es) | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| US20140359799A1 (en) | 2011-12-23 | 2014-12-04 | Case Western Reserve University | Targeted gene modification using hybrid recombinant adeno-associated virus |
| US9644205B2 (en) | 2012-04-25 | 2017-05-09 | The Regents Of The University Of California | Synthetic promoter for modulating gene expression |
| EP2900686B1 (en) | 2012-09-28 | 2020-06-10 | The University of North Carolina At Chapel Hill | Aav vectors targeted to oligodendrocytes |
| US9938541B2 (en) | 2012-12-25 | 2018-04-10 | Takara Bio Inc. | AAV variant |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| ES2926774T3 (es) | 2013-03-15 | 2022-10-28 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores AAV de doble unión de glicano |
| EP3003391B1 (en) | 2013-05-31 | 2021-09-22 | The Regents of The University of California | Adeno-associated virus variants and methods of use thereof |
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| WO2015164757A1 (en) | 2014-04-25 | 2015-10-29 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| LT3137497T (lt) | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| KR101902343B1 (ko) | 2014-07-11 | 2018-09-28 | 후아웨이 테크놀러지 컴퍼니 리미티드 | 네트워크 장치 및 사용자 장치, 및 그 방법들 |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
| CN107073051B (zh) | 2014-10-21 | 2021-08-24 | 马萨诸塞大学 | 重组aav变体及其用途 |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| EP4344741A3 (en) | 2014-11-21 | 2024-08-28 | The University of North Carolina at Chapel Hill | Aav vectors targeted to the central nervous system |
| US20180030096A1 (en) | 2015-02-03 | 2018-02-01 | University Of Florida Research Foundation, Inc. | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof |
| AU2016219789B2 (en) | 2015-02-20 | 2021-03-25 | Fondazione Telethon | Methods and compositions for treating genetic eye diseases |
| CN106032540B (zh) | 2015-03-16 | 2019-10-25 | 中国科学院上海生命科学研究院 | CRISPR/Cas9核酸内切酶体系的腺相关病毒载体构建及其用途 |
| 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 |
| WO2016164642A1 (en) | 2015-04-08 | 2016-10-13 | The United States Of America, As Represented By The Secretary Of Health And Human Services | Viral gene therapy as treatment for cholesterol storage disease or disorder |
| WO2017015102A1 (en) | 2015-07-17 | 2017-01-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for achieving high levels of transduction in human liver cells |
| ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| WO2017066764A2 (en) | 2015-10-16 | 2017-04-20 | William Marsh Rice University | Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses |
| WO2017083722A1 (en) | 2015-11-11 | 2017-05-18 | Greenberg Kenneth P | Crispr compositions and methods of using the same for gene therapy |
| US10240145B2 (en) | 2015-11-25 | 2019-03-26 | The Board Of Trustees Of The Leland Stanford Junior University | CRISPR/Cas-mediated genome editing to treat EGFR-mutant lung cancer |
| KR102423442B1 (ko) | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| CN106884014B (zh) | 2015-12-16 | 2020-11-13 | 北京五加和基因科技有限公司 | 腺相关病毒反向末端重复序列突变体及其应用 |
| JP6947739B2 (ja) | 2016-02-16 | 2021-10-13 | ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー | 既存のヒト中和抗体に対して耐性である新規組換えアデノ随伴ウイルスカプシド |
| PL3423154T3 (pl) | 2016-03-01 | 2021-11-02 | Intraop Medical Corporation | System napromieniania wiązką elektronową o niskiej energii, który generuje wiązki elektronowe z precyzyjnie kontrolowaną i regulowlaną głębokością penetracji, przydatny w zastosowaniach terapeutycznych |
| AU2017302013B2 (en) | 2016-07-29 | 2022-05-26 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| US20190262373A1 (en) | 2016-08-16 | 2019-08-29 | The University Of North Carolina At Chapel Hill | Methods and compositions for targeted gene transfer |
| US20200316221A1 (en) | 2016-10-13 | 2020-10-08 | University Of Massachusetts | Aav capsid designs |
| WO2018119330A2 (en) | 2016-12-22 | 2018-06-28 | Oregon Health & Science University | Adeno associated viral vectors |
| CN108330147A (zh) | 2017-01-20 | 2018-07-27 | 上海吉凯基因化学技术有限公司 | 一种重组腺相关病毒载体生产工艺的建立 |
| US11680275B2 (en) * | 2017-06-06 | 2023-06-20 | University Of Massachusetts | Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome |
| PE20200488A1 (es) | 2017-06-27 | 2020-03-03 | Regeneron Pharma | Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas |
| EP3645021A4 (en) | 2017-06-30 | 2021-04-21 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
| EP3808849A1 (en) | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| RU2770922C2 (ru) | 2017-09-20 | 2022-04-25 | 4Д Молекьюлар Терапьютикс Инк. | Капсиды вариантов аденоассоциированных вирусов и методы их применения |
| WO2019068854A1 (en) | 2017-10-06 | 2019-04-11 | Ospedale San Raffaele S.R.L. | GENE THERAPY OF NEURODEGENERATIVE DISEASES USING VAA VECTORS |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| EP3759218A4 (en) | 2018-02-28 | 2021-12-08 | The University of North Carolina at Chapel Hill | PROCEDURES AND COMPOSITIONS FOR ANTIBODY ELIMINATING VIRUS VECTORS |
| WO2019213668A1 (en) | 2018-05-04 | 2019-11-07 | Oregon Health & Science University | Human anti-aav2 capsid polyclonal antibody epitopes |
| AU2018422759B2 (en) * | 2018-05-07 | 2026-01-22 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| US11821009B2 (en) | 2018-05-15 | 2023-11-21 | Cornell University | Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery |
| BR112020023298A2 (pt) | 2018-05-15 | 2021-03-09 | University Of Washington | composições e métodos para reduzir a spliceopatia e tratamento de distúrbios de dominância de rna |
| WO2019222329A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| US20210207167A1 (en) | 2018-05-16 | 2021-07-08 | Voyager Therapeutics, Inc. | Aav serotypes for brain specific payload delivery |
| US20210214749A1 (en) | 2018-05-16 | 2021-07-15 | Voyager Therapeutics, Inc. | Directed evolution |
| 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 |
| WO2020028751A2 (en) * | 2018-08-03 | 2020-02-06 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
| 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 |
| EP3856762A1 (en) * | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CN113166208B (zh) * | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| US20210371470A1 (en) | 2018-10-12 | 2021-12-02 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| JP2022513618A (ja) | 2018-11-20 | 2022-02-09 | 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 | 血液脳関門の標的化に有用な組成物および方法 |
| WO2020132455A1 (en) | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2020154324A1 (en) | 2019-01-22 | 2020-07-30 | Ix Layer Inc. | Systems and methods for access management and clustering of genomic or phenotype data |
| US20220143214A1 (en) | 2019-01-30 | 2022-05-12 | The Broad Institute, Inc. | Systems for evolved adeno-associated viruses (aavs) for targeted delivery |
| WO2020160508A1 (en) | 2019-01-31 | 2020-08-06 | Oregon Health & Science University | Methods for using transcription-dependent directed evolution of aav capsids |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| 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 |
| EP3947700A4 (en) | 2019-04-01 | 2023-01-04 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| 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 |
| JP2022526018A (ja) | 2019-04-11 | 2022-05-20 | カリフォルニア インスティテュート オブ テクノロジー | 脳内への特異性が増強されたウイルス組成物 |
| JP2022530457A (ja) | 2019-04-26 | 2022-06-29 | サンガモ セラピューティクス， インコーポレイテッド | 遺伝子操作ａａｖ |
| TW202532428A (zh) | 2019-04-26 | 2025-08-16 | 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 |
| WO2020223280A1 (en) | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
| WO2021025995A1 (en) | 2019-08-02 | 2021-02-11 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
| CN118221785A (zh) | 2019-10-16 | 2024-06-21 | 上海药明康德新药开发有限公司 | 新的aav变体 |
| KR20220110263A (ko) | 2019-12-04 | 2022-08-05 | 더 보드 오브 트러스티스 오브 더 리랜드 스탠포드 쥬니어 유니버시티 | 내인성 조절인자의 표적화에 의한 혈액-뇌 장벽 약물 수송의 향상 |
| WO2021202651A1 (en) | 2020-04-01 | 2021-10-07 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| US12552838B2 (en) | 2020-04-20 | 2026-02-17 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| WO2021222831A2 (en) | 2020-05-01 | 2021-11-04 | The Broad Institute, Inc. | Engineered central nervous system compositions |
| JP2023524577A (ja) | 2020-05-05 | 2023-06-12 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 改変アデノ随伴ウイルス５カプシドおよびその使用 |
| TWI900553B (zh) | 2020-05-13 | 2025-10-11 | 美商航海家醫療公司 | Aav蛋白殼之趨性重定向 |
| CN116390934A (zh) | 2020-05-26 | 2023-07-04 | 塑造治疗公司 | 功能性aav衣壳的高通量工程化 |
| AU2021329527A1 (en) | 2020-08-21 | 2023-03-23 | Capsida, Inc. | Adeno-associated virus compositions having preferred expression levels |
| WO2022076750A2 (en) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns or muscle delivery |
| WO2022187473A2 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| WO2022187548A1 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| JP2024513949A (ja) | 2021-04-13 | 2024-03-27 | カプシダ， インコーポレイテッド | 脳内発現レベルが高いａａｖ組成物 |
| EP4322974A4 (en) | 2021-04-13 | 2025-06-25 | Capsida, Inc. | Adeno-associated virus compositions having increased brain enrichment |
| WO2022221421A2 (en) | 2021-04-13 | 2022-10-20 | Capsida, Inc. | Aav compositions with high brain expression for treating mucopolysaccharidosis ii |
| US20250281643A1 (en) | 2021-04-13 | 2025-09-11 | Capsida, Inc. | Selected aav compositions having preferred brain enrichment |
| EP4334332A4 (en) | 2021-05-04 | 2025-11-12 | California Inst Of Techn | VAA RECOMMENDANTS FOR ADMINISTRATION TO THE CENTRAL NERVOUS SYSTEM AND CEREBRAL VASCULATURE |
| US20230047753A1 (en) | 2021-07-23 | 2023-02-16 | California Institute Of Technology | Viral vectors for enhanced ultrasound-mediated delivery to the brain |
| US20230086710A1 (en) | 2021-09-14 | 2023-03-23 | California Institute Of Technology | Aav capsid variants |
| WO2023044483A2 (en) | 2021-09-20 | 2023-03-23 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| EP4405373A4 (en) | 2021-09-21 | 2026-02-11 | California Inst Of Techn | VIRAL COMPOUNDS TO TARGET THE NERVOUS SYSTEM AND THE LUNGS |
| EP4426716A1 (en) | 2021-11-02 | 2024-09-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2023091934A1 (en) | 2021-11-16 | 2023-05-25 | California Institute Of Technology | Adeno-associated virus compositions having preferred heart and skeletal muscle enrichment |
| US20250049955A1 (en) | 2021-11-17 | 2025-02-13 | Voyager Therapeutics, Inc. | Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| US20250034559A1 (en) | 2021-11-17 | 2025-01-30 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of tau-related disorders |
| WO2023092002A2 (en) | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis and disorders associatedwith the spinal cord |
| EP4433169A1 (en) | 2021-11-17 | 2024-09-25 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| CA3249551A1 (en) | 2022-02-08 | 2023-08-17 | Voyager Therapeutics, Inc. | AAV Capsid Variants and Their Uses |
| JP2025508968A (ja) | 2022-03-03 | 2025-04-10 | カリフォルニア インスティテュート オブ テクノロジー | 血液脳関門を横断するための組成物および方法 |
| CA3254570A1 (en) | 2022-03-24 | 2023-09-28 | Capsida, Inc. | ADENE-ASSOCIATED VIRUS COMPOSITIONS WITH INCREASED CARDIAC ENRICHEMENT |
| CA3254573A1 (en) | 2022-03-24 | 2023-09-28 | Capsida, Inc. | SELECTED VAA COMPOSITIONS WITH A PREFERRED BRAIN ENRICHEMENT |
| WO2023201207A1 (en) | 2022-04-11 | 2023-10-19 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| EP4519284A2 (en) | 2022-05-06 | 2025-03-12 | Capsida, Inc. | Adeno-associated virus compositions having increased brain enrichment |
| WO2023220695A2 (en) | 2022-05-13 | 2023-11-16 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| CA3256915A1 (en) | 2022-05-17 | 2023-11-23 | Capsida, Inc. | SELECTED ADENE-ASSOCIATED VIRUS COMPOSITIONS HAVING PREFERRED LEVELS OF CEREBRAL, SPINAL CORD AND/OR CARDIAC EXPRESSION |
| EP4532515A1 (en) | 2022-06-02 | 2025-04-09 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
| US20250255989A1 (en) | 2022-06-16 | 2025-08-14 | Capsida, Inc | Adeno-associated virus compositions having increased brain enrichment and/or heart enrichment |
| US20250346637A1 (en) | 2022-06-16 | 2025-11-13 | Capsida, Inc. | Adeno-associated virus compositions having increased brain enrichment and decreased liver enrichment |
| EP4543473A1 (en) | 2022-06-22 | 2025-04-30 | Voyager Therapeutics, Inc. | Tau binding compounds |
| CA3259902A1 (en) | 2022-06-28 | 2024-01-04 | Voyager Therapeutics, Inc. | AAV Capsid Variants and Their Uses |
| US20250388626A1 (en) | 2022-07-06 | 2025-12-25 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| JP2025528068A (ja) | 2022-08-03 | 2025-08-26 | ボイジャー セラピューティクス インコーポレイテッド | 血液脳関門を通過させるための組成物及び方法 |
| KR20250069606A (ko) | 2022-09-15 | 2025-05-19 | 보이저 테라퓨틱스, 인크. | 타우 결합 화합물 |
| WO2024086747A1 (en) | 2022-10-19 | 2024-04-25 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| EP4605538A2 (en) | 2022-10-19 | 2025-08-27 | Capsida Biotherapeutics, Inc. | Adeno-associated virus compositions having preferredbrain enrichment and low liver enrichment |
| WO2024092164A1 (en) | 2022-10-27 | 2024-05-02 | California Institute Of Technology | Targets for receptor-mediated control of therapeutic biodistribution and efficacy |
| AU2023378899A1 (en) | 2022-11-13 | 2025-06-26 | Alexion Pharma International Operations Limited | Gene therapy for frontotemporal dementia |
| WO2024191778A1 (en) | 2023-03-10 | 2024-09-19 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2024226761A2 (en) | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| WO2024226790A1 (en) | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| AU2024266140A1 (en) | 2023-05-02 | 2025-09-11 | Voyager Therapeutics, Inc. | Compositions and methods for regulating mapt |
| WO2024229161A1 (en) | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency |
| EP4705484A1 (en) | 2023-05-03 | 2026-03-11 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of tau-related disorders |
| EP4705487A2 (en) | 2023-05-03 | 2026-03-11 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to frataxin deficiency |
| AU2024266519A1 (en) | 2023-05-03 | 2025-12-04 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency |
| KR20260021096A (ko) | 2023-05-03 | 2026-02-12 | 보이저 테라퓨틱스, 인크. | Cdkl5 결핍과 관련된 장애의 치료를 위한 조성물 및 방법 |
| WO2024229173A2 (en) | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to ataxin-2 |
| WO2024229164A2 (en) | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| 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 |
| AU2024271436A1 (en) | 2023-05-15 | 2025-11-13 | Sangamo Therapeutics, Inc. | Engineered blood brain barrier penetrant aav capsids |
| AU2024274127A1 (en) | 2023-05-15 | 2025-11-13 | Sangamo Therapeutics, Inc. | Fitness maturation of engineered aav capsid stac-102 |
| TW202523846A (zh) | 2023-08-16 | 2025-06-16 | 美商航海家醫療公司 | 用於治療與共濟蛋白(frataxin)不足有關之疾病之組合物及方法 |
| WO2025038796A1 (en) | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to cdkl5 deficiency |
| WO2025038805A1 (en) | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency |
| WO2025038795A1 (en) | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025038430A1 (en) | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2025038802A1 (en) | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to ataxin-2 |
| WO2025147436A1 (en) | 2024-01-03 | 2025-07-10 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
-
2021
- 2021-03-31 TW TW110111995A patent/TWI900553B/zh active
- 2021-03-31 MX MX2022014256A patent/MX2022014256A/es unknown
- 2021-03-31 CN CN202180049682.3A patent/CN116096734A/zh active Pending
- 2021-03-31 JP JP2022568852A patent/JP7731909B2/ja active Active
- 2021-03-31 EP EP21720894.1A patent/EP4149955A1/en active Pending
- 2021-03-31 WO PCT/US2021/025061 patent/WO2021230987A1/en not_active Ceased
- 2021-03-31 KR KR1020227043585A patent/KR20230022175A/ko active Pending
- 2021-03-31 PE PE2022002647A patent/PE20230767A1/es unknown
- 2021-03-31 BR BR112022023106A patent/BR112022023106A2/pt unknown
- 2021-03-31 US US17/924,800 patent/US20230203102A1/en active Pending
- 2021-03-31 IL IL298001A patent/IL298001A/en unknown
- 2021-03-31 AU AU2021273447A patent/AU2021273447B2/en active Active
- 2021-03-31 CA CA3182970A patent/CA3182970A1/en active Pending
- 2021-10-26 US US17/511,482 patent/US11859200B2/en active Active
-
2022
- 2022-11-11 CO CONC2022/0016156A patent/CO2022016156A2/es unknown
-
2023
- 2023-11-16 US US18/511,290 patent/US12577588B2/en active Active
-
2025
- 2025-08-20 JP JP2025136742A patent/JP2025173511A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CO2022016156A2 (es) | 2023-01-26 |
| US20240200097A1 (en) | 2024-06-20 |
| US20220042044A1 (en) | 2022-02-10 |
| TW202208397A (zh) | 2022-03-01 |
| EP4149955A1 (en) | 2023-03-22 |
| US12577588B2 (en) | 2026-03-17 |
| US11859200B2 (en) | 2024-01-02 |
| WO2021230987A1 (en) | 2021-11-18 |
| AU2021273447A1 (en) | 2022-12-08 |
| MX2022014256A (es) | 2023-01-11 |
| JP2025173511A (ja) | 2025-11-27 |
| CN116096734A (zh) | 2023-05-09 |
| JP2023525810A (ja) | 2023-06-19 |
| AU2021273447B2 (en) | 2026-03-05 |
| JP7731909B2 (ja) | 2025-09-01 |
| IL298001A (en) | 2023-01-01 |
| KR20230022175A (ko) | 2023-02-14 |
| PE20230767A1 (es) | 2023-05-09 |
| US20230203102A1 (en) | 2023-06-29 |
| TWI900553B (zh) | 2025-10-11 |
| BR112022023106A2 (pt) | 2023-01-17 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CA3182970A1 (en) | Redirection of tropism of aav capsids | |
| US11932668B2 (en) | Selective recovery | |
| US20230287451A1 (en) | Novel aav capsids and compositions containing same | |
| CA3061368A1 (en) | Compositions and methods of treating huntington's disease | |
| CN116096377A (zh) | 跨物种相容的腺相关病毒组合物及其使用方法 | |
| KR102917057B1 (ko) | 뇌에 지향성을 가지는 aav 돌연변이체 | |
| KR20260011702A (ko) | 조작된 aav 캡시드 stac-102의 적합성 성숙 | |
| AU2021255934A1 (en) | Compositions and methods for treatment of neurological disorders | |
| CN121358754A (zh) | 具有外周器官去靶向性的新型嗜神经性腺相关病毒衣壳 | |
| EP4409008A1 (en) | Slc13a5 gene therapy vectors and uses thereof | |
| US20250197456A1 (en) | Novel aav capsids for targeting nervous system and uses thereof | |
| HK40085441A (en) | Redirection of tropism of aav capsids | |
| WO2025235689A9 (en) | Peptides targeting alpl for blood-brain barrier crossing | |
| WO2025235666A1 (en) | Peptides targeting ldlr for blood-brain barrier crossing | |
| WO2025235682A1 (en) | Peptides targeting tfrc for blood-brain barrier crossing | |
| HK40016209A (en) | Selective recovery |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| D11 | Substantive examination requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED Effective date: 20250321 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - VOLUNTARY AMENDMENT Effective date: 20250321 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20250321 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 4TH ANNIV.) - STANDARD Year of fee payment: 4 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20250324 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT Effective date: 20250324 Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20250324 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20250401 |
|
| D00 | Search and/or examination requested or commenced |
Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D00-D118 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION REQUIREMENTS DETERMINED COMPLIANT Effective date: 20250414 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT Effective date: 20250414 |
|
| P13 | Application amended |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED Effective date: 20250414 |
|
| D11 | Substantive examination requested |
Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-D155 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALL REQUIREMENTS FOR EXAMINATION DETERMINED COMPLIANT Effective date: 20250429 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20250516 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 5TH ANNIV.) - STANDARD Year of fee payment: 5 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20260324 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20260324 |