JP2016538885A5 - - Google Patents
Download PDFInfo
- 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 (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| EP3215191B1 (en) | 2014-11-05 | 2024-08-14 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| CN114717264A (zh) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| EP3236984B1 (en) | 2014-12-23 | 2020-07-22 | The U.S.A. 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 |
| EP3387434A1 (en) * | 2015-12-11 | 2018-10-17 | The U.S.A. As Represented By The Secretary, Department Of Health And Human Services | Gene therapy for combined methylmalonic acidemia/aciduria and hyperhomocysteinemia/homocystinuria, cobalamin c type, and deficiency of mmachc |
| EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| US10584337B2 (en) | 2016-05-18 | 2020-03-10 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| IL297576B2 (en) | 2016-05-18 | 2024-02-01 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| EP3831281B1 (en) | 2016-08-30 | 2026-02-25 | The Regents of The University of California | Devices and systems for biomedical targeting and delivery |
| AU2018212002A1 (en) * | 2017-01-30 | 2019-08-29 | The United States Of America, 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 |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| MX2019013172A (es) | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
| 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 |
| 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. |
| 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 |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| WO2019172286A1 (ja) | 2018-03-09 | 2019-09-12 | 第一三共株式会社 | 糖原病Ｉａ型治療薬 |
| CN112424359A (zh) | 2018-05-15 | 2021-02-26 | 沃雅戈治疗公司 | 用于治疗帕金森氏病的组合物和方法 |
| SG11202011191WA (en) * | 2018-05-16 | 2020-12-30 | Spark Therapeutics Inc | Codon-optimized acid αlpha-glucosidase expression cassettes and methods of using same |
| EP3814499A2 (en) | 2018-06-28 | 2021-05-05 | CRISPR Therapeutics AG | Compositions and methods for genomic editing by insertion of donor polynucleotides |
| JP2022500436A (ja) * | 2018-09-13 | 2022-01-04 | モダーナティエックス・インコーポレイテッドModernaTX, Inc. | 糖原病を処置するためのグルコース−６−ホスファターゼをコードするポリヌクレオチド |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | 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 |
| CA3123841A1 (en) * | 2018-12-18 | 2020-06-25 | 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 |
| KR102398032B1 (ko) * | 2020-09-22 | 2022-05-19 | 비피진 주식회사 | 암 치료를 위한 5''-뉴클레오티다아제 변이체 |
| 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 |
| JP4559429B2 (ja) | 2003-05-21 | 2010-10-06 | ジェンザイム・コーポレーション | 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法 |
| 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 |
| 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 |
| EP2529020B1 (en) | 2010-01-28 | 2018-04-25 | The Children's Hospital of Philadelphia | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
-
2014
- 2014-11-25 JP JP2016554828A patent/JP6649265B2/ja active Active
- 2014-11-25 EP EP18186594.0A patent/EP3415620B1/en active Active
- 2014-11-25 US US15/038,979 patent/US9644216B2/en active Active
- 2014-11-25 ES ES14812375.5T patent/ES2690643T3/es active Active
- 2014-11-25 MX MX2016006774A patent/MX375360B/es active IP Right Grant
- 2014-11-25 CN CN201480074046.6A patent/CN105934515B/zh active Active
- 2014-11-25 CA CA2930872A patent/CA2930872C/en active Active
- 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
-
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