JP2016538885A5 - - Google Patents
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- 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.)
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- 239000000203 mixture Substances 0.000 claims description 19
- 150000007523 nucleic acids Chemical class 0.000 claims description 12
- 108020004707 nucleic acids Proteins 0.000 claims description 12
- 239000002773 nucleotide Substances 0.000 claims description 8
- 125000003729 nucleotide group Chemical group 0.000 claims description 8
- 206010018464 Glycogen storage disease type I Diseases 0.000 claims description 4
- 208000002555 Hepatorenal form of glycogen storage disease Diseases 0.000 claims description 4
- 241000432074 Adeno-associated virus Species 0.000 claims description 2
- 229920001850 Nucleic acid sequence Polymers 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
- 230000003612 virological Effects 0.000 claims description 2
- 238000002560 therapeutic procedure Methods 0.000 claims 1
- 208000007345 Glycogen Storage Disease Diseases 0.000 description 3
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 (US07794700-20100914-C00152.png) | 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)
Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| BR112017009497A2 (pt) | 2014-11-05 | 2018-02-06 | Voyager Therapeutics, Inc. | polinucleotídeos de aadc para o tratamento da doença de parkinson |
| ES2878451T3 (es) | 2014-11-14 | 2021-11-18 | Voyager Therapeutics Inc | Polinucleótidos moduladores |
| US10597660B2 (en) | 2014-11-14 | 2020-03-24 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| EP3230441A4 (en) | 2014-12-12 | 2018-10-03 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| ES2824829T3 (es) | 2014-12-23 | 2021-05-13 | Us Health | Vectores de virus adenoasociado que codifican G6PC modificada y usos de estos |
| CN109328231A (zh) * | 2015-11-06 | 2019-02-12 | 克里斯普治疗股份公司 | 用于治疗1a型糖原贮积病的材料和方法 |
| 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 |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| US11951121B2 (en) | 2016-05-18 | 2024-04-09 | Voyager Therapeutics, Inc. | Compositions and methods for treating Huntington's disease |
| CA3035522A1 (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 |
| EP3574104A1 (en) * | 2017-01-30 | 2019-12-04 | 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 |
| JP7048574B2 (ja) * | 2017-03-10 | 2022-04-05 | 国立研究開発法人国立成育医療研究センター | アンチセンスオリゴヌクレオチドおよび糖原病Ｉａ型予防または治療用組成物 |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| AU2018302016A1 (en) | 2017-07-17 | 2020-02-06 | The Regents Of The University Of California | Trajectory array guide system |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| TWI804518B (zh) | 2017-10-16 | 2023-06-11 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| CN115976028A (zh) | 2018-03-09 | 2023-04-18 | 第一三共株式会社 | 糖原病Ia型治疗药 |
| JP2021523718A (ja) * | 2018-05-16 | 2021-09-09 | スパーク セラピューティクス インコーポレイテッドＳｐａｒｋ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． | コドン最適化された酸性α−グルコシダーゼ発現カセット及びそれを使用する方法 |
| SG11202012499RA (en) | 2018-06-28 | 2021-01-28 | Crispr Therapeutics Ag | Compositions and methods for genomic editing by insertion of donor polynucleotides |
| EP3894066A4 (en) * | 2018-12-12 | 2022-09-07 | Merck Sharp & Dohme Corp. | CELLULAR BIO-IDENTITY TEST FOR INSULIN |
| US20220017922A1 (en) * | 2018-12-18 | 2022-01-20 | Ultragenyx Pharmaceutical Inc. | Methods and compositions for treating glycogen storage diseases |
| 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 |
| EP2281877A3 (en) | 2003-05-21 | 2011-06-01 | Genzyme Corporation | Methods for producing preparations of recombinant AAV virions substantially free of empty capsids |
| AU2009209408B2 (en) | 2008-01-29 | 2015-06-11 | Beacon Therapeutics Limited | Recombinant virus production using mammalian cells in suspension |
| WO2010109053A1 (es) * | 2009-03-27 | 2010-09-30 | Proyeto De Biomedicina Cima, S.L. | Métodos y composiciones para el tratamiento de cirrosis y fibrosis hepática |
| US8679837B2 (en) | 2009-04-02 | 2014-03-25 | University Of Florida Research Foundation, Inc. | Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors |
| DK2529020T3 (en) | 2010-01-28 | 2018-08-06 | Childrens Hospital Philadelphia | SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY |
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2014
- 2014-11-25 WO PCT/US2014/067415 patent/WO2015081101A1/en active Application Filing
- 2014-11-25 EP EP18186594.0A patent/EP3415620B1/en active Active
- 2014-11-25 ES ES14812375.5T patent/ES2690643T3/es active Active
- 2014-11-25 MX MX2016006774A patent/MX2016006774A/es active IP Right Grant
- 2014-11-25 US US15/038,979 patent/US9644216B2/en active Active
- 2014-11-25 AU AU2014354839A patent/AU2014354839B2/en active Active
- 2014-11-25 EP EP14812375.5A patent/EP3074510B1/en active Active
- 2014-11-25 CA CA2930872A patent/CA2930872C/en active Active
- 2014-11-25 CN CN201480074046.6A patent/CN105934515B/zh active Active
- 2014-11-25 JP JP2016554828A patent/JP6649265B2/ja active Active
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2016
- 2016-05-16 IL IL245659A patent/IL245659B/en active IP Right Grant
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2017
- 2017-04-21 US US15/493,622 patent/US10113183B2/en active Active
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2018
- 2018-10-01 US US16/148,435 patent/US11060110B2/en active Active
