CN107532177A - 腺相关病毒变体及其使用方法 - Google Patents
腺相关病毒变体及其使用方法 Download PDFInfo
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- CN107532177A CN107532177A CN201680022686.1A CN201680022686A CN107532177A CN 107532177 A CN107532177 A CN 107532177A CN 201680022686 A CN201680022686 A CN 201680022686A CN 107532177 A CN107532177 A CN 107532177A
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- 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
- C12N15/861—Adenoviral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P35/00—Antineoplastic agents
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- C07—ORGANIC CHEMISTRY
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- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- 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
- C12N15/74—Vectors or expression systems specially adapted for prokaryotic hosts other than E. coli, e.g. Lactobacillus, Micromonospora
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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- 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
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- 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
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- Health & Medical Sciences (AREA)
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- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
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- Molecular Biology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
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- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562137580P | 2015-03-24 | 2015-03-24 | |
| US62/137,580 | 2015-03-24 | ||
| PCT/US2016/023822 WO2016154344A1 (en) | 2015-03-24 | 2016-03-23 | Adeno-associated virus variants and methods of use thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN107532177A true CN107532177A (zh) | 2018-01-02 |
Family
ID=56978687
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201680022686.1A Pending CN107532177A (zh) | 2015-03-24 | 2016-03-23 | 腺相关病毒变体及其使用方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US10883117B2 (enExample) |
| EP (1) | EP3277819B1 (enExample) |
| JP (1) | JP6836999B2 (enExample) |
| CN (1) | CN107532177A (enExample) |
| AU (1) | AU2016235163B2 (enExample) |
| CA (1) | CA2979065A1 (enExample) |
| ES (1) | ES2860712T3 (enExample) |
| MX (1) | MX388331B (enExample) |
| WO (1) | WO2016154344A1 (enExample) |
Cited By (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110295151A (zh) * | 2019-07-17 | 2019-10-01 | 博鑫仪器(天津)有限公司 | 一类腺依赖病毒病毒重组质粒、重组病毒及构建方法 |
| CN110950934A (zh) * | 2019-12-31 | 2020-04-03 | 复旦大学 | 一种腺相关病毒衣壳蛋白、载体及其构建方法与应用 |
| CN111909246A (zh) * | 2019-05-08 | 2020-11-10 | 中国科学院脑科学与智能技术卓越创新中心 | 高效感染支持细胞的aav突变体 |
| CN112041442A (zh) * | 2018-02-28 | 2020-12-04 | 北卡罗来纳大学查佩尔希尔分校 | 用于逃避抗体的病毒载体的方法和组合物 |
| CN112226461A (zh) * | 2020-08-21 | 2021-01-15 | 华侨大学 | Cd4阳性细胞特异性基因传递载体及其应用 |
| CN112424217A (zh) * | 2018-05-14 | 2021-02-26 | 生物马林药物股份有限公司 | 肝脏靶向的腺病毒相关病毒载体 |
| CN112566923A (zh) * | 2018-06-12 | 2021-03-26 | 北卡罗来纳大学教堂山分校 | 合成嗜肝性腺相关病毒衣壳及其用途 |
| WO2021073568A1 (en) * | 2019-10-16 | 2021-04-22 | Wuxi Apptec (Shanghai) Co., Ltd. | A novel aav variant |
| CN113121654A (zh) * | 2021-04-19 | 2021-07-16 | 信念医药科技(上海)有限公司 | 新型腺相关病毒衣壳蛋白及包含其的新型腺相关病毒载体 |
| CN113121653A (zh) * | 2021-04-19 | 2021-07-16 | 上海信致医药科技有限公司 | 肌肉和视网膜特异性的新型腺相关病毒衣壳蛋白 |
| CN113717248A (zh) * | 2020-09-30 | 2021-11-30 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN114502575A (zh) * | 2019-06-10 | 2022-05-13 | 同源药物公司 | 用于arsa基因转移的腺相关病毒组合物和其使用方法 |
| CN114729384A (zh) * | 2019-09-12 | 2022-07-08 | 博德研究所 | 工程化腺相关病毒衣壳 |
| CN115087738A (zh) * | 2019-08-29 | 2022-09-20 | 小利兰·斯坦福大学托管委员会 | 用于穿越人血脑屏障的腺相关病毒载体 |
| CN115551878A (zh) * | 2020-05-01 | 2022-12-30 | 波士顿学院董事会 | 用于增强的基因疗法的腺相关病毒(aav)的受控修饰 |
| WO2023108507A1 (en) * | 2021-12-15 | 2023-06-22 | National Institute Of Biological Sciences, Beijing | Recombinant aav vectors and use thereof |
| EP4244360A4 (en) * | 2020-11-16 | 2025-05-07 | Beijing SoloBio Genetechnology Company Ltd. | Recombinant adeno-associated viruses with enhanced liver tropism and uses thereof |
| US12410447B2 (en) | 2019-10-16 | 2025-09-09 | The Broad Institute, Inc. | Engineered muscle targeting compositions |
Families Citing this family (62)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20160369298A1 (en) | 2013-09-26 | 2016-12-22 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| JP6689249B2 (ja) | 2014-03-17 | 2020-05-20 | 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 | 錐体細胞における増強された遺伝子発現のための組成物および方法 |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| RU2716422C2 (ru) | 2014-11-14 | 2020-03-11 | Вояджер Терапьютикс, Инк. | Композиции и способы лечения бокового амиотрофического склероза (als) |
| CN112410339A (zh) | 2014-11-14 | 2021-02-26 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| HK1245326A1 (zh) | 2014-12-12 | 2018-08-24 | Voyager Therapeutics, Inc. | 用於生产scaav的组合物和方法 |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| CN108137655B (zh) | 2015-09-28 | 2022-04-22 | 北卡罗来纳-查佩尔山大学 | 逃避抗体的病毒载体的方法和组合物 |
| US12188037B2 (en) | 2015-10-22 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Synthetic combinatorial AAV3 capsid library |
| US10983110B2 (en) | 2015-12-02 | 2021-04-20 | Voyager Therapeutics, Inc. | Assays for the detection of AAV neutralizing antibodies |
| WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| CA3252099A1 (en) | 2016-05-18 | 2025-06-05 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| CN109831916B (zh) | 2016-05-18 | 2023-07-21 | 沃雅戈治疗公司 | 治疗亨廷顿氏舞蹈病的组合物和方法 |
| KR20230039779A (ko) | 2016-07-29 | 2023-03-21 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법 |
| EP3506817A4 (en) | 2016-08-30 | 2020-07-22 | The Regents of The University of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| EP3585883A4 (en) | 2017-02-21 | 2021-04-14 | University of Florida Research Foundation, Incorporated | MODIFIED AAV CAPSID PROTEINS AND USES THEREOF |
| JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | 筋萎縮性側索硬化症(als)治療組成物および方法 |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | ハンチントン病治療組成物および方法 |
| WO2018211409A1 (en) | 2017-05-15 | 2018-11-22 | University Of Florida Research Foundation, Inc. | Core master regulators of glioblastoma stem cells |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| WO2019006182A1 (en) | 2017-06-30 | 2019-01-03 | The Regents Of The University Of California | VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF |
| WO2019018342A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | NETWORK EQUIPMENT TRACK GUIDE SYSTEM |
| KR102850887B1 (ko) | 2017-08-03 | 2025-08-28 | 보이저 테라퓨틱스, 인크. | Aav의 전달을 위한 조성물 및 방법 |
| NZ763018A (en) * | 2017-09-20 | 2023-02-24 | 4D Molecular Therapeutics Inc | Adeno-associated virus variant capsids and methods of use thereof |
| MX2020003042A (es) | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| CN119242711A (zh) | 2017-10-16 | 2025-01-03 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| SG11202009451VA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Antibody-evading virus vectors |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| JP7406677B2 (ja) | 2018-04-03 | 2023-12-28 | ギンコ バイオワークス インコーポレイテッド | 抗体を回避するウイルスベクター |
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| 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 |
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| US12331320B2 (en) | 2018-10-10 | 2025-06-17 | The Research Foundation For The State University Of New York | Genome edited cancer cell vaccines |
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| US20220154217A1 (en) | 2019-04-01 | 2022-05-19 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| US20230137971A1 (en) | 2019-07-11 | 2023-05-04 | Tenaya Therapeutics Inc. | Cardiac cell reprogramming with micrornas and other factors |
| JP2022544237A (ja) * | 2019-08-09 | 2022-10-17 | ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. | ヒト神経膠芽腫細胞をターゲティングするためのaavカプシドバリアント |
| TW202128736A (zh) | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療c型尼曼—匹克病之腺相關病毒載體 |
| KR20220139956A (ko) | 2020-02-13 | 2022-10-17 | 테나야 테라퓨틱스, 인코포레이티드 | 심장 질환을 치료하기 위한 유전자 요법 벡터 |
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| CN118510905A (zh) * | 2021-12-06 | 2024-08-16 | 学校法人顺天堂 | 改变型腺伴随病毒载体 |
| TW202421787A (zh) | 2022-09-06 | 2024-06-01 | 美商特納亞治療股份有限公司 | 保護心臟之心臟病療法 |
| CN117304256B (zh) * | 2022-12-08 | 2024-04-16 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2024215655A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Cardioprotective bag3 therapies |
| WO2024215653A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Guide rnas, vectors, and virions for targeting mutations in the pln gene |
| AU2024271436A1 (en) * | 2023-05-15 | 2025-11-13 | Sangamo Therapeutics, Inc. | Engineered blood brain barrier penetrant aav capsids |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
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| US20180066285A1 (en) | 2018-03-08 |
| EP3277819A1 (en) | 2018-02-07 |
| WO2016154344A1 (en) | 2016-09-29 |
| ES2860712T3 (es) | 2021-10-05 |
| JP2018514190A (ja) | 2018-06-07 |
| HK1246355A1 (zh) | 2018-09-07 |
| EP3277819A4 (en) | 2018-09-05 |
| CA2979065A1 (en) | 2016-09-29 |
| AU2016235163B2 (en) | 2022-03-24 |
| EP3277819B1 (en) | 2021-03-03 |
| AU2016235163A1 (en) | 2017-10-05 |
| MX388331B (es) | 2025-03-19 |
| MX2017012097A (es) | 2018-06-15 |
| US10883117B2 (en) | 2021-01-05 |
| US20210147876A1 (en) | 2021-05-20 |
| JP6836999B2 (ja) | 2021-03-03 |
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