TWI910162B - 跨物種相容腺相關病毒組合物及其使用方法 - Google Patents
跨物種相容腺相關病毒組合物及其使用方法Info
- Publication number
- TWI910162B TWI910162B TW110116293A TW110116293A TWI910162B TW I910162 B TWI910162 B TW I910162B TW 110116293 A TW110116293 A TW 110116293A TW 110116293 A TW110116293 A TW 110116293A TW I910162 B TWI910162 B TW I910162B
- Authority
- TW
- Taiwan
- Prior art keywords
- aav
- capsid protein
- vector
- amino acid
- capsid
- Prior art date
Links
Classifications
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Urology & Nephrology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063020062P | 2020-05-05 | 2020-05-05 | |
| US63/020,062 | 2020-05-05 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| TW202208631A TW202208631A (zh) | 2022-03-01 |
| TWI910162B true TWI910162B (zh) | 2026-01-01 |
Family
ID=78468375
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TW110116293A TWI910162B (zh) | 2020-05-05 | 2021-05-05 | 跨物種相容腺相關病毒組合物及其使用方法 |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US12577586B2 (enExample) |
| EP (1) | EP4125977A4 (enExample) |
| JP (1) | JP7756439B2 (enExample) |
| KR (1) | KR20230029616A (enExample) |
| CN (1) | CN116096377B (enExample) |
| AR (1) | AR122013A1 (enExample) |
| AU (1) | AU2021268946A1 (enExample) |
| BR (1) | BR112022022644A2 (enExample) |
| CA (1) | CA3177869A1 (enExample) |
| IL (1) | IL297577A (enExample) |
| MX (1) | MX2022013963A (enExample) |
| TW (1) | TWI910162B (enExample) |
| WO (1) | WO2021226267A2 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TWI910162B (zh) * | 2020-05-05 | 2026-01-01 | 美國公爵大學 | 跨物種相容腺相關病毒組合物及其使用方法 |
| KR20240095539A (ko) | 2021-10-08 | 2024-06-25 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 변이체 및 이의 사용 방법 |
| WO2024044725A2 (en) * | 2022-08-24 | 2024-02-29 | Regenxbio Inc. | Recombinant adeno-associated viruses and uses thereof |
| WO2024168089A2 (en) * | 2023-02-07 | 2024-08-15 | Duke University | Compositions comprising aavs and methods for treating gastrointestinal diseases |
| US20240261438A1 (en) * | 2023-02-08 | 2024-08-08 | Ginkgo Bioworks, Inc. | Gene therapy for angelman syndrome |
| 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 |
| EP4689139A1 (en) * | 2023-03-31 | 2026-02-11 | Duke University | Compositions comprising brain-tropic aavs and methods of use thereof |
| KR20260082361A (ko) | 2023-08-31 | 2026-06-04 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 폴리펩타이드 및 이의 사용 방법 |
| 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 |
| WO2025250454A1 (en) * | 2024-05-28 | 2025-12-04 | University Of Rochester | Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo |
| 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 |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20150126588A1 (en) * | 2012-05-09 | 2015-05-07 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| US20190060425A1 (en) * | 2017-06-15 | 2019-02-28 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| CN110913881A (zh) * | 2017-03-14 | 2020-03-24 | 加利福尼亚大学董事会 | 工程化crispr cas9免疫隐身 |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| EP1135468B1 (en) | 1998-11-10 | 2010-01-06 | University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
| US7314912B1 (en) | 1999-06-21 | 2008-01-01 | Medigene Aktiengesellschaft | AAv scleroprotein, production and use thereof |
| US7465583B2 (en) | 2000-06-01 | 2008-12-16 | The University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| CN101528916B (zh) * | 2006-04-28 | 2013-09-04 | 宾夕法尼亚大学托管会 | 规模可调的aav生产方法 |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| CA2826273C (en) | 2011-02-10 | 2021-11-02 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| US11136557B2 (en) * | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| US10746742B2 (en) * | 2014-04-25 | 2020-08-18 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| IL314171A (en) | 2015-09-28 | 2024-09-01 | Univ Florida | Methods and compositions for antibody-evading virus vectors |
| EP3583220A4 (en) * | 2017-02-15 | 2021-07-07 | The University of North Carolina at Chapel Hill | PROCEDURES AND COMPOSITIONS FOR GENERAL TRANSFER IN THE VASCULAR SYSTEM |
| JP2020510424A (ja) | 2017-02-28 | 2020-04-09 | アドヴェラム バイオテクノロジーズ， インコーポレイテッド | 改変ａａｖカプシドおよびその使用 |
| WO2018170310A1 (en) | 2017-03-15 | 2018-09-20 | The University Of North Carolina At Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| MX2020010465A (es) * | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| CA3095179A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| WO2019222444A2 (en) | 2018-05-16 | 2019-11-21 | Voyager Therapeutics, Inc. | Directed evolution |
| EP3860659A1 (en) * | 2018-10-01 | 2021-08-11 | The Trustees of the University of Pennsylvania | Compositions useful for treating gm1 gangliosidosis |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| TWI910162B (zh) | 2020-05-05 | 2026-01-01 | 美國公爵大學 | 跨物種相容腺相關病毒組合物及其使用方法 |
-
2021
- 2021-05-05 TW TW110116293A patent/TWI910162B/zh active
- 2021-05-05 MX MX2022013963A patent/MX2022013963A/es unknown
- 2021-05-05 EP EP21800135.2A patent/EP4125977A4/en active Pending
- 2021-05-05 AU AU2021268946A patent/AU2021268946A1/en active Pending
- 2021-05-05 JP JP2022567079A patent/JP7756439B2/ja active Active
- 2021-05-05 BR BR112022022644A patent/BR112022022644A2/pt unknown
- 2021-05-05 CA CA3177869A patent/CA3177869A1/en active Pending
- 2021-05-05 KR KR1020227042650A patent/KR20230029616A/ko active Pending
- 2021-05-05 CN CN202180044147.9A patent/CN116096377B/zh active Active
- 2021-05-05 WO PCT/US2021/030937 patent/WO2021226267A2/en not_active Ceased
- 2021-05-05 AR ARP210101222A patent/AR122013A1/es not_active Application Discontinuation
- 2021-05-05 IL IL297577A patent/IL297577A/en unknown
- 2021-05-05 US US17/922,962 patent/US12577586B2/en active Active
-
2023
- 2023-09-26 US US18/474,533 patent/US12037598B2/en active Active
- 2023-09-26 US US18/474,506 patent/US12146151B2/en active Active
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20150126588A1 (en) * | 2012-05-09 | 2015-05-07 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| CN110913881A (zh) * | 2017-03-14 | 2020-03-24 | 加利福尼亚大学董事会 | 工程化crispr cas9免疫隐身 |
| US20190060425A1 (en) * | 2017-06-15 | 2019-02-28 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
Also Published As
| Publication number | Publication date |
|---|---|
| IL297577A (en) | 2022-12-01 |
| JP2023524291A (ja) | 2023-06-09 |
| MX2022013963A (es) | 2023-02-22 |
| TW202208631A (zh) | 2022-03-01 |
| AR122013A1 (es) | 2022-08-03 |
| EP4125977A2 (en) | 2023-02-08 |
| US12146151B2 (en) | 2024-11-19 |
| US20230151389A1 (en) | 2023-05-18 |
| CN116096377A (zh) | 2023-05-09 |
| CA3177869A1 (en) | 2021-11-11 |
| KR20230029616A (ko) | 2023-03-03 |
| US12577586B2 (en) | 2026-03-17 |
| US12037598B2 (en) | 2024-07-16 |
| US20240076695A1 (en) | 2024-03-07 |
| US20240067988A1 (en) | 2024-02-29 |
| WO2021226267A3 (en) | 2021-12-16 |
| BR112022022644A2 (pt) | 2023-01-17 |
| WO2021226267A2 (en) | 2021-11-11 |
| JP7756439B2 (ja) | 2025-10-20 |
| AU2021268946A1 (en) | 2022-12-01 |
| CN116096377B (zh) | 2026-01-02 |
| EP4125977A4 (en) | 2024-09-25 |
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