UY36611A - Producción de vectores adeno-asociados de gran tamaño - Google Patents
Producción de vectores adeno-asociados de gran tamañoInfo
- Publication number
- UY36611A UY36611A UY0001036611A UY36611A UY36611A UY 36611 A UY36611 A UY 36611A UY 0001036611 A UY0001036611 A UY 0001036611A UY 36611 A UY36611 A UY 36611A UY 36611 A UY36611 A UY 36611A
- Authority
- UY
- Uruguay
- Prior art keywords
- adeno
- vectors
- associate
- production
- aav
- Prior art date
Links
- 239000013598 vector Substances 0.000 title 1
- 239000002245 particle Substances 0.000 abstract 3
- 239000013607 AAV vector Substances 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
Classifications
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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
- 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
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/745—Blood coagulation or fibrinolysis factors
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
-
- 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
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10041—Use of virus, viral particle or viral elements as a 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
- C12N2750/14143—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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Hematology (AREA)
- Toxicology (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Marine Sciences & Fisheries (AREA)
- Physical Education & Sports Medicine (AREA)
- Pulmonology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
Se proporcionan aquí métodos para producir una partícula de virus adenoasociado (AAV) que contiene un genoma de AAV recombinante de gran tamaño (por ejemplo, más de 4,7 kb). En algunos aspectos, la invención proporciona partículas AAV y vectores de AAV que comprenden genomas de rAAV de gran tamaño. También se proporcionan líneas celulares productoras para producir partículas AAV que comprenden genomas de gran tamaño.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562144862P | 2015-04-08 | 2015-04-08 | |
| US201562220067P | 2015-09-17 | 2015-09-17 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| UY36611A true UY36611A (es) | 2016-11-30 |
Family
ID=56943906
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| UY0001036611A UY36611A (es) | 2015-04-08 | 2016-04-08 | Producción de vectores adeno-asociados de gran tamaño |
Country Status (27)
| Country | Link |
|---|---|
| US (1) | US10815497B2 (es) |
| EP (2) | EP3280799B1 (es) |
| JP (3) | JP6878299B2 (es) |
| KR (2) | KR20250126875A (es) |
| CN (1) | CN107864657A (es) |
| AU (3) | AU2016245806A1 (es) |
| BR (1) | BR112017021505A2 (es) |
| CA (1) | CA2982123A1 (es) |
| CL (1) | CL2017002537A1 (es) |
| CO (1) | CO2017011344A2 (es) |
| CR (1) | CR20170505A (es) |
| DK (1) | DK3280799T3 (es) |
| EA (1) | EA201792236A1 (es) |
| EC (1) | ECSP17074016A (es) |
| ES (1) | ES3008382T3 (es) |
| GT (1) | GT201700214A (es) |
| HU (1) | HUE069779T2 (es) |
| IL (3) | IL283291B (es) |
| MX (1) | MX2017012935A (es) |
| PE (1) | PE20171800A1 (es) |
| PH (1) | PH12017501836A1 (es) |
| PL (1) | PL3280799T3 (es) |
| SG (3) | SG11201708203UA (es) |
| TN (1) | TN2017000431A1 (es) |
| TW (1) | TWI707951B (es) |
| UY (1) | UY36611A (es) |
| WO (1) | WO2016164609A2 (es) |
Families Citing this family (47)
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| FI3889173T3 (fi) | 2013-02-15 | 2023-10-02 | Bioverativ Therapeutics Inc | Optimoitu tekijä viii:n geeni |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2016004113A1 (en) | 2014-06-30 | 2016-01-07 | Biogen Ma Inc. | Optimized factor ix gene |
| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| IL292999A (en) | 2014-11-14 | 2022-07-01 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN112410338A (zh) | 2014-11-14 | 2021-02-26 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| TWI707951B (zh) * | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| PE20181338A1 (es) | 2015-09-24 | 2018-08-21 | Biomarin Pharm Inc | Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden |
| KR20180118659A (ko) | 2016-02-01 | 2018-10-31 | 바이오버라티브 테라퓨틱스 인크. | 최적화된 viii 인자 유전자 |
| US11326182B2 (en) | 2016-04-29 | 2022-05-10 | 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 |
| KR20220108216A (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| SG11201809699XA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| CN110650673B (zh) | 2016-08-30 | 2024-04-09 | 加利福尼亚大学董事会 | 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统 |
| US11518991B2 (en) | 2017-02-24 | 2022-12-06 | Georg-August-Universitaet Goettingen Stiftung Oeffentlichen Rechts, Universitaetsmedizin | Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated RASAL1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including RASAL1 in a subject |
| 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 |
| SG11201909870SA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN118956965A (zh) * | 2017-05-19 | 2024-11-15 | 编码治疗公司 | 高活性调控元件 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| TWI832036B (zh) | 2017-08-03 | 2024-02-11 | 美商航海家醫療公司 | 用於aav之遞送之組合物及方法 |
| BR112020002394A2 (pt) | 2017-08-09 | 2020-07-28 | Bioverativ Therapeutics Inc. | moléculas de ácidos nucleicos e usos das mesmas |
| 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. |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| JP7401432B2 (ja) | 2017-12-01 | 2023-12-19 | エンコーデッド セラピューティクス, インコーポレイテッド | 操作されたdna結合タンパク質 |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| US20210338838A1 (en) * | 2018-07-20 | 2021-11-04 | University Of Utah Research Foundation | Gene therapy for macular degeneration |
| KR102925832B1 (ko) | 2018-08-09 | 2026-02-11 | 바이오버라티브 테라퓨틱스 인크. | 핵산 분자 및 비바이러스 유전자 치료를 위한 이의 용도 |
| CN113383010B (zh) | 2018-09-28 | 2025-09-09 | 沃雅戈治疗公司 | 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法 |
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| EP4121567B1 (en) * | 2020-03-16 | 2025-12-31 | The University of North Carolina at Chapel Hill | COMPOSITIONS AND METHODS FOR THE SELECTIVE DETECTION OF TUMOR-DERIVED VIRAL DNA |
| KR20230074703A (ko) | 2020-06-24 | 2023-05-31 | 바이오버라티브 테라퓨틱스 인크. | 유리 인자 viii을 상기 단백질이 발현되도록 변형된 렌티바이러스 벡터의 제제로부터 제거하는 방법 |
| JP2023536148A (ja) | 2020-07-30 | 2023-08-23 | シェイプ セラピューティクス インク. | Raavウイルス粒子の誘導産生のための安定な細胞株 |
| US20220090130A1 (en) | 2020-08-23 | 2022-03-24 | Bioverativ Therapeutics Inc. | MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA) |
| JP2024534123A (ja) | 2021-08-23 | 2024-09-18 | バイオベラティブ セラピューティクス インコーポレイテッド | バキュロウイルス発現システム |
| AR126845A1 (es) | 2021-08-23 | 2023-11-22 | Bioverativ Therapeutics Inc | Producción de adn de extremo cerrado con secuencias repetidas de terminal invertida |
| IL310997A (en) | 2021-08-23 | 2024-04-01 | Bioverativ Therapeutics Inc | Factor VIII gene optimization |
| EP4408874A1 (en) | 2021-09-30 | 2024-08-07 | Bioverativ Therapeutics Inc. | Nucleic acids encoding factor viii polypeptides with reduced immunogenicity |
| US12234472B2 (en) | 2021-10-18 | 2025-02-25 | Regeneron Pharmaceuticals, Inc. | Eukaryotic cells comprising adenovirus-associated virus polynucleotides |
| US20240392318A1 (en) * | 2023-04-26 | 2024-11-28 | Sanofi | Host cell lines and methods for identifying and using such host cell lines |
| WO2026083329A1 (en) | 2024-10-18 | 2026-04-23 | Precision Biosciences, Inc. | Methods of nuclease-initiated homology directed repair and replacement and compositions and uses thereof |
| WO2026083328A1 (en) | 2024-10-18 | 2026-04-23 | Precision Biosciences, Inc. | Base editing nucleotide sequences using homology directed repair |
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| JP2001500015A (ja) | 1996-09-06 | 2001-01-09 | 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 | T7ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法 |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| EP2369002A1 (en) | 1999-08-09 | 2011-09-28 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
| WO2001027303A1 (en) | 1999-10-12 | 2001-04-19 | The University Of North Carolina At Chapel Hill | Adeno-associated virus vectors encoding factor viii and methods of using the same |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
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-
2016
- 2016-04-06 TW TW105110751A patent/TWI707951B/zh active
- 2016-04-07 TN TNP/2017/000431A patent/TN2017000431A1/en unknown
- 2016-04-07 CN CN201680033119.6A patent/CN107864657A/zh active Pending
- 2016-04-07 US US15/565,020 patent/US10815497B2/en active Active
- 2016-04-07 SG SG11201708203UA patent/SG11201708203UA/en unknown
- 2016-04-07 SG SG10201912912QA patent/SG10201912912QA/en unknown
- 2016-04-07 BR BR112017021505A patent/BR112017021505A2/pt not_active Application Discontinuation
- 2016-04-07 EA EA201792236A patent/EA201792236A1/ru unknown
- 2016-04-07 KR KR1020257027291A patent/KR20250126875A/ko active Pending
- 2016-04-07 CA CA2982123A patent/CA2982123A1/en active Pending
- 2016-04-07 PL PL16767050.4T patent/PL3280799T3/pl unknown
- 2016-04-07 AU AU2016245806A patent/AU2016245806A1/en not_active Abandoned
- 2016-04-07 IL IL283291A patent/IL283291B/en unknown
- 2016-04-07 EP EP16767050.4A patent/EP3280799B1/en active Active
- 2016-04-07 DK DK16767050.4T patent/DK3280799T3/da active
- 2016-04-07 SG SG10201909381W patent/SG10201909381WA/en unknown
- 2016-04-07 MX MX2017012935A patent/MX2017012935A/es unknown
- 2016-04-07 PE PE2017001997A patent/PE20171800A1/es unknown
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