JP2016538885A5 - - Google Patents
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- Publication number
- JP2016538885A5 JP2016538885A5 JP2016554828A JP2016554828A JP2016538885A5 JP 2016538885 A5 JP2016538885 A5 JP 2016538885A5 JP 2016554828 A JP2016554828 A JP 2016554828A JP 2016554828 A JP2016554828 A JP 2016554828A JP 2016538885 A5 JP2016538885 A5 JP 2016538885A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- raav
- vector
- seq
- nucleic acid
- 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.)
- Granted
Links
- 239000000203 mixture Substances 0.000 claims description 19
- 239000013598 vector Substances 0.000 claims description 14
- 150000007523 nucleic acids Chemical class 0.000 claims description 12
- 102000039446 nucleic acids Human genes 0.000 claims description 12
- 108020004707 nucleic acids Proteins 0.000 claims description 12
- 239000002773 nucleotide Substances 0.000 claims description 10
- 125000003729 nucleotide group Chemical group 0.000 claims description 10
- 208000011460 glycogen storage disease due to glucose-6-phosphatase deficiency type IA Diseases 0.000 claims description 5
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 4
- 206010018464 Glycogen storage disease type I Diseases 0.000 claims description 4
- 239000013607 AAV vector Substances 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 238000001990 intravenous administration Methods 0.000 claims description 2
- 239000002245 particle Substances 0.000 claims description 2
- 239000013647 rAAV8 vector Substances 0.000 claims description 2
- 230000003612 virological effect Effects 0.000 claims description 2
- 238000002560 therapeutic procedure Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 8
- 208000007345 glycogen storage disease Diseases 0.000 description 2
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361908861P | 2013-11-26 | 2013-11-26 | |
| US61/908,861 | 2013-11-26 | ||
| PCT/US2014/067415 WO2015081101A1 (en) | 2013-11-26 | 2014-11-25 | Adeno-associated virus vectors for treatment of glycogen storage disease |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016538885A JP2016538885A (ja) | 2016-12-15 |
| JP2016538885A5 true JP2016538885A5 (enExample) | 2017-11-24 |
| JP6649265B2 JP6649265B2 (ja) | 2020-02-19 |
Family
ID=52101617
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016554828A Active JP6649265B2 (ja) | 2013-11-26 | 2014-11-25 | 糖原病の処置のためのアデノ随伴ウイルスベクター |
Country Status (10)
| Country | Link |
|---|---|
| US (3) | US9644216B2 (enExample) |
| EP (2) | EP3415620B1 (enExample) |
| JP (1) | JP6649265B2 (enExample) |
| CN (1) | CN105934515B (enExample) |
| AU (1) | AU2014354839B2 (enExample) |
| CA (1) | CA2930872C (enExample) |
| ES (1) | ES2690643T3 (enExample) |
| IL (1) | IL245659B (enExample) |
| MX (1) | MX375360B (enExample) |
| WO (1) | WO2015081101A1 (enExample) |
Families Citing this family (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US10415044B2 (en) | 2014-12-23 | 2019-09-17 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Adeno-associated virus vectors encoding modified G6PC and uses thereof |
| US11866727B2 (en) | 2015-11-06 | 2024-01-09 | Crispr Therapeutics Ag | Materials and methods for treatment of glycogen storage disease type 1A |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| 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 |
| CA3252099A1 (en) | 2016-05-18 | 2025-06-05 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| 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 |
| US20190367944A1 (en) * | 2017-01-30 | 2019-12-05 | The U.S.A., As Represented By The Secretary, Department Of Health And Human Services | Recombinant virus vectors for the treatment of glycogen storage disease |
| EP3594346A4 (en) | 2017-03-10 | 2020-12-16 | National Center For Child Health And Development | ANTISENS OLIGONUCLEOTIDE AND COMPOSITION FOR THE PREVENTION OR TREATMENT OF TYPE IA GLYCOGEN STORAGE DISEASE |
| 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 |
| WO2019018342A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | NETWORK EQUIPMENT TRACK GUIDE SYSTEM |
| 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. |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| 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 |
| US11958878B2 (en) | 2018-03-09 | 2024-04-16 | Daiichi Sankyo Company, Limited | Therapeutic agent for glycogen storage disease type IA |
| BR112020023082A2 (pt) | 2018-05-15 | 2021-02-09 | Voyager Therapeutics, Inc. | composições e métodos para o tratamento de doença de parkinson |
| MY207355A (en) * | 2018-05-16 | 2025-02-21 | Spark Therapeutics Inc | Codon-optimized acid lpha-glucosidase expression cassettes and methods of using same |
| JP7550057B2 (ja) | 2018-06-28 | 2024-09-12 | クリスパー セラピューティクス アーゲー | ドナーポリヌクレオチドの挿入によるゲノム編集のための組成物および方法 |
| WO2020056147A2 (en) * | 2018-09-13 | 2020-03-19 | Modernatx, Inc. | Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease |
| CA3114621A1 (en) | 2018-09-28 | 2020-04-02 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| WO2020123269A1 (en) * | 2018-12-12 | 2020-06-18 | Merck Sharp & Dohme Corp. | Cell-based bioidentity test for insulin |
| ES2969222T3 (es) * | 2018-12-18 | 2024-05-17 | Ultragenyx Pharmaceutical Inc | Métodos y composiciones para el tratamiento de enfermedades del almacenamiento de glucógeno |
| EP3913060A1 (en) * | 2020-05-22 | 2021-11-24 | Genethon | Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy |
| CN111972355A (zh) * | 2020-08-31 | 2020-11-24 | 中国医学科学院北京协和医院 | GSDIa型糖原累积症小鼠模型及其构建方法 |
| WO2023069891A1 (en) * | 2021-10-19 | 2023-04-27 | University Of Connecticut | Compositions and methods for the treatment of glycogen storage disorders |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6759050B1 (en) | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
| DK2277996T3 (da) | 2003-05-21 | 2014-10-20 | Genzyme Corp | Fremgangsmåder til fremstilling af præparationer af rekombinante aav-virioner, der i hovedsagen er fri for tomme capsider |
| EP2242840B1 (en) | 2008-01-29 | 2019-07-24 | Applied Genetic Technologies Corporation | Recombinant adeno-associated virus production using bhk cells in suspension |
| AU2010227419A1 (en) * | 2009-03-27 | 2011-10-20 | Proyecto De Biomedicina Cima, S.L. | Methods and compositions for the treatment of cirrhosis and liver fibrosis |
| WO2010114948A2 (en) | 2009-04-02 | 2010-10-07 | University Of Florida Research Foundation, Inc. | An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors |
| MX340102B (es) | 2010-01-28 | 2016-06-24 | The Children's Hospital Of Philadelphia * | Una plataforma de fabricacion ajustable en escala, para la purificacion de vector viral y vectores virales asi purificados para el uso en terapia de genes. |
-
2014
- 2014-11-25 CA CA2930872A patent/CA2930872C/en active Active
- 2014-11-25 ES ES14812375.5T patent/ES2690643T3/es active Active
- 2014-11-25 JP JP2016554828A patent/JP6649265B2/ja active Active
- 2014-11-25 EP EP18186594.0A patent/EP3415620B1/en active Active
- 2014-11-25 MX MX2016006774A patent/MX375360B/es active IP Right Grant
- 2014-11-25 EP EP14812375.5A patent/EP3074510B1/en active Active
- 2014-11-25 WO PCT/US2014/067415 patent/WO2015081101A1/en not_active Ceased
- 2014-11-25 AU AU2014354839A patent/AU2014354839B2/en active Active
- 2014-11-25 CN CN201480074046.6A patent/CN105934515B/zh active Active
- 2014-11-25 US US15/038,979 patent/US9644216B2/en active Active
-
2016
- 2016-05-16 IL IL245659A patent/IL245659B/en active IP Right Grant
-
2017
- 2017-04-21 US US15/493,622 patent/US10113183B2/en active Active
-
2018
- 2018-10-01 US US16/148,435 patent/US11060110B2/en active Active
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