CA2878401C - Modified adeno-associated virus vector compositions - Google Patents
Modified adeno-associated virus vector compositions Download PDFInfo
- Publication number
- CA2878401C CA2878401C CA2878401A CA2878401A CA2878401C CA 2878401 C CA2878401 C CA 2878401C CA 2878401 A CA2878401 A CA 2878401A CA 2878401 A CA2878401 A CA 2878401A CA 2878401 C CA2878401 C CA 2878401C
- Authority
- CA
- Canada
- Prior art keywords
- aav
- nucleic acid
- vector
- sequence
- seq
- 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.)
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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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- 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
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261668839P | 2012-07-06 | 2012-07-06 | |
| US61/668,839 | 2012-07-06 | ||
| PCT/US2013/031644 WO2014007858A1 (en) | 2012-07-06 | 2013-03-14 | Modified adeno-associated virus vector compositions |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2878401A1 CA2878401A1 (en) | 2014-01-09 |
| CA2878401C true CA2878401C (en) | 2022-07-19 |
Family
ID=49882407
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2878401A Active CA2878401C (en) | 2012-07-06 | 2013-03-14 | Modified adeno-associated virus vector compositions |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US9540659B2 (https=) |
| EP (1) | EP2870237B1 (https=) |
| JP (1) | JP6366581B2 (https=) |
| CN (1) | CN104619832B (https=) |
| AU (1) | AU2013287261B2 (https=) |
| BR (1) | BR112015000161A2 (https=) |
| CA (1) | CA2878401C (https=) |
| ES (1) | ES2703177T3 (https=) |
| HK (1) | HK1210214A1 (https=) |
| IN (1) | IN2015KN00074A (https=) |
| RU (1) | RU2679843C2 (https=) |
| WO (1) | WO2014007858A1 (https=) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2679843C2 (ru) * | 2012-07-06 | 2019-02-13 | Юниверсити Оф Айова Рисерч Фаундейшн | Векторные композиции с модифицированным аденоассоциированным вирусом |
| EP3146051B8 (en) * | 2014-05-20 | 2019-11-27 | University of Iowa Research Foundation | Huntington's disease therapeutic compounds |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| US20190000940A1 (en) * | 2015-07-31 | 2019-01-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of aadc deficiency |
| WO2017161273A1 (en) * | 2016-03-18 | 2017-09-21 | The Children's Hospital Of Philadelphia | Therapeutic for treatment of diseases including the central nervous system |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| KR102427379B1 (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| 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 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CA3070087A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| 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) |
| EP3724326A1 (en) * | 2017-12-11 | 2020-10-21 | Editas Medicine, Inc. | Cpf1-related methods and compositions for gene editing |
| US20190256867A1 (en) | 2018-02-01 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| JP7244547B2 (ja) | 2018-02-19 | 2023-03-22 | ホモロジー・メディシンズ・インコーポレイテッド | Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| AU2019247748A1 (en) | 2018-04-03 | 2020-10-08 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| KR20210019996A (ko) | 2018-05-15 | 2021-02-23 | 보이저 테라퓨틱스, 인크. | 파킨슨병의 치료를 위한 조성물 및 방법 |
| US20210254103A1 (en) * | 2018-07-02 | 2021-08-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| CA3140049A1 (en) | 2019-05-14 | 2020-11-19 | Duke University | Compositions and methods for the treatment of atpase-mediated diseases |
| EP3999647A1 (en) * | 2019-07-18 | 2022-05-25 | Lysogene | Compositions and methods for the treatment of sanfilippo disease and other disorders |
| JP2022551739A (ja) | 2019-10-17 | 2022-12-13 | ストライドバイオ，インコーポレイテッド | ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター |
| US12611436B2 (en) | 2019-10-17 | 2026-04-28 | Sarepta Therapeutics, Inc. | AAV transfer cassette |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| CN115768900A (zh) * | 2020-05-11 | 2023-03-07 | 戈迪安生物技术公司 | 病毒递送媒剂选择 |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| AU2021328475A1 (en) | 2020-08-19 | 2023-03-16 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| JP2024506296A (ja) * | 2021-02-05 | 2024-02-13 | メイズ セラピューティクス， インコーポレイテッド | スタッファーポリヌクレオチド配列を含むベクター |
| US20240218397A1 (en) * | 2021-05-04 | 2024-07-04 | Regenxbio Inc. | Novel aav vectors and methods and uses thereof |
| GB2623164A (en) * | 2022-08-09 | 2024-04-10 | Univ Sheffield | Viral vector |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) * | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| JPH1033175A (ja) * | 1996-07-24 | 1998-02-10 | Hisamitsu Pharmaceut Co Inc | 組換えアデノ随伴ウイルスベクターの製造方法 |
| US6171855B1 (en) * | 1998-05-28 | 2001-01-09 | The Regents Of The University Of Michigan | Vectors |
| US6943153B1 (en) * | 1999-03-15 | 2005-09-13 | The Regents Of The University Of California | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
| WO2001032899A1 (en) * | 1999-10-29 | 2001-05-10 | Takara Shuzo Co., Ltd. | Gene transfer method |
| US7132277B1 (en) * | 2000-01-31 | 2006-11-07 | Merck & Co., Inc. | Helper dependent vector system for gene therapy |
| US7387896B2 (en) | 2003-03-26 | 2008-06-17 | The Regents Of The University Of Michigan | MicroRNA vectors |
| BRPI0415563A (pt) * | 2003-10-20 | 2007-01-02 | Nsgene As | método para o tratamento do mal de parkinson, uso de um vetor viral, vetor de expressão viral, composição farmacêutica, célula hospedeira isolada, linhagem de células de empacotamento, mamìfero não humano quimérico, dispositivo de cultura celular implantável, cápsula biocompatìvel, método para tratamento de uma doença do sistema nervoso, célula de mamìfero, e, método para produzir neurturin ou um seu equivalente funcional |
| WO2009134681A2 (en) * | 2008-04-30 | 2009-11-05 | The Trustees Of The University Of Pennsylvania | Aav7 viral vectors for targeted delivery of rpe cells |
| US9546369B2 (en) * | 2010-04-23 | 2017-01-17 | University Of Massachusetts | Multicistronic expression constructs |
| WO2012109667A1 (en) | 2011-02-12 | 2012-08-16 | University Of Iowa Research Foundation | Therapeutic compounds |
| RU2679843C2 (ru) * | 2012-07-06 | 2019-02-13 | Юниверсити Оф Айова Рисерч Фаундейшн | Векторные композиции с модифицированным аденоассоциированным вирусом |
-
2013
- 2013-03-14 RU RU2015103901A patent/RU2679843C2/ru active
- 2013-03-14 AU AU2013287261A patent/AU2013287261B2/en active Active
- 2013-03-14 JP JP2015520166A patent/JP6366581B2/ja active Active
- 2013-03-14 WO PCT/US2013/031644 patent/WO2014007858A1/en not_active Ceased
- 2013-03-14 EP EP13812649.5A patent/EP2870237B1/en active Active
- 2013-03-14 CN CN201380035802.XA patent/CN104619832B/zh active Active
- 2013-03-14 IN IN74KON2015 patent/IN2015KN00074A/en unknown
- 2013-03-14 BR BR112015000161A patent/BR112015000161A2/pt not_active Application Discontinuation
- 2013-03-14 CA CA2878401A patent/CA2878401C/en active Active
- 2013-03-14 HK HK15110883.2A patent/HK1210214A1/xx unknown
- 2013-03-14 ES ES13812649T patent/ES2703177T3/es active Active
- 2013-03-14 US US14/410,983 patent/US9540659B2/en active Active
-
2017
- 2017-07-07 US US15/644,491 patent/US20170306355A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| HK1210214A1 (en) | 2016-04-15 |
| RU2015103901A (ru) | 2016-08-27 |
| AU2013287261B2 (en) | 2019-03-07 |
| US9540659B2 (en) | 2017-01-10 |
| CN104619832A (zh) | 2015-05-13 |
| CA2878401A1 (en) | 2014-01-09 |
| EP2870237A1 (en) | 2015-05-13 |
| JP2015522280A (ja) | 2015-08-06 |
| AU2013287261A1 (en) | 2015-01-22 |
| US20150184197A1 (en) | 2015-07-02 |
| US20170306355A1 (en) | 2017-10-26 |
| BR112015000161A2 (pt) | 2017-06-27 |
| JP6366581B2 (ja) | 2018-08-08 |
| WO2014007858A1 (en) | 2014-01-09 |
| IN2015KN00074A (https=) | 2015-07-31 |
| ES2703177T3 (es) | 2019-03-07 |
| CN104619832B (zh) | 2021-03-16 |
| EP2870237B1 (en) | 2018-09-26 |
| EP2870237A4 (en) | 2015-12-23 |
| RU2679843C2 (ru) | 2019-02-13 |
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