JP2021522173A5 - - Google Patents
Info
- Publication number
- JP2021522173A5 JP2021522173A5 JP2020557222A JP2020557222A JP2021522173A5 JP 2021522173 A5 JP2021522173 A5 JP 2021522173A5 JP 2020557222 A JP2020557222 A JP 2020557222A JP 2020557222 A JP2020557222 A JP 2020557222A JP 2021522173 A5 JP2021522173 A5 JP 2021522173A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- composition according
- subject
- virus
- composition
- Prior art date
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862659281P | 2018-04-18 | 2018-04-18 | |
| US62/659,281 | 2018-04-18 | ||
| PCT/US2019/028108 WO2019204593A1 (en) | 2018-04-18 | 2019-04-18 | Gene therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2021522173A JP2021522173A (ja) | 2021-08-30 |
| JP2021522173A5 true JP2021522173A5 (https=) | 2022-04-18 |
| JPWO2019204593A5 JPWO2019204593A5 (https=) | 2022-04-18 |
| JP7769355B2 JP7769355B2 (ja) | 2025-11-13 |
Family
ID=68239912
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020557222A Active JP7769355B2 (ja) | 2018-04-18 | 2019-04-18 | ミトコンドリアｄｎａ枯渇症候群を含む不均衡ヌクレオチドプールに起因する疾患の遺伝子治療 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US12419970B2 (https=) |
| EP (1) | EP3781671A4 (https=) |
| JP (1) | JP7769355B2 (https=) |
| KR (1) | KR20210009317A (https=) |
| CN (1) | CN112312929A (https=) |
| AU (1) | AU2019255745A1 (https=) |
| BR (1) | BR112020021324A2 (https=) |
| CA (1) | CA3097392A1 (https=) |
| CL (1) | CL2020002704A1 (https=) |
| CO (1) | CO2020014193A2 (https=) |
| IL (1) | IL278130B1 (https=) |
| MX (1) | MX2020010994A (https=) |
| WO (1) | WO2019204593A1 (https=) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9549909B2 (en) | 2013-05-03 | 2017-01-24 | The Katholieke Universiteit Leuven | Method for the treatment of dravet syndrome |
| PT4293009T (pt) | 2015-12-22 | 2025-09-08 | Zogenix International Ltd | Composições de fenfluramina e métodos de preparação das mesmas |
| WO2017112701A1 (en) | 2015-12-22 | 2017-06-29 | Zogenix International Limited | Metabolism resistant fenfluramine analogs and methods of using the same |
| AU2017315273B2 (en) | 2016-08-24 | 2021-04-29 | Zogenix International Limited | Formulation for inhibiting formation of 5-HT 2B agonists and methods of using same |
| US10682317B2 (en) | 2017-09-26 | 2020-06-16 | Zogenix International Limited | Ketogenic diet compatible fenfluramine formulation |
| EP3790537A1 (en) | 2018-05-11 | 2021-03-17 | Zogenix International Limited | Compositions and methods for treating seizure-induced sudden death |
| AU2019384963B2 (en) | 2018-11-19 | 2022-05-26 | Zogenix International Limited | Methods of treating Rett syndrome using fenfluramine |
| US20220403417A1 (en) * | 2019-11-20 | 2022-12-22 | University Of Massachusetts | Aav-based delivery of thymine kinase 2 |
| US11612574B2 (en) | 2020-07-17 | 2023-03-28 | Zogenix International Limited | Method of treating patients infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) |
| US20220096514A1 (en) * | 2020-09-29 | 2022-03-31 | Zogenix International Limited | Methods of treating thymidine kinase 2 deficiency by administering deoxycytidine and deoxythymidine |
| EP4313068A4 (en) | 2021-03-26 | 2024-12-25 | UCB Biosciences, Inc. | Aqueous solutions containing purines and pyrimidines and uses thereof |
| KR20250004463A (ko) * | 2023-06-30 | 2025-01-08 | 사회복지법인 삼성생명공익재단 | Hydra NDi1가 탑재된 재조합 아데노부속바이러스 벡터를 포함하는, 미토콘드리아 복합체 1 이상질환의 예방, 개선 또는 치료용 조성물 |
| CN120060299B (zh) * | 2025-02-21 | 2025-11-28 | 三峡大学 | 一种密码子优化的twnk基因、腺相关病毒载体及其应用 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5252334A (en) | 1989-09-08 | 1993-10-12 | Cygnus Therapeutic Systems | Solid matrix system for transdermal drug delivery |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5843742A (en) | 1994-12-16 | 1998-12-01 | Avigen Incorporated | Adeno-associated derived vector systems for gene delivery and integration into target cells |
| US5697899A (en) | 1995-02-07 | 1997-12-16 | Gensia | Feedback controlled drug delivery system |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved AAV vectors for gene therapy |
| US5656016A (en) | 1996-03-18 | 1997-08-12 | Abbott Laboratories | Sonophoretic drug delivery system |
| US5797898A (en) | 1996-07-02 | 1998-08-25 | Massachusetts Institute Of Technology | Microchip drug delivery devices |
| US5783208A (en) | 1996-07-19 | 1998-07-21 | Theratech, Inc. | Transdermal drug delivery matrix for coadministering estradiol and another steroid |
| US5779708A (en) | 1996-08-15 | 1998-07-14 | Cyberdent, Inc. | Intraosseous drug delivery device and method |
| AU722624B2 (en) | 1996-09-06 | 2000-08-10 | Trustees Of The University Of Pennsylvania, The | An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| CN101460055A (zh) | 2006-04-07 | 2009-06-17 | 得克萨斯大学体系董事会 | 和腺伴随病毒-噬菌体颗粒相关的方法和组合物 |
| FR3004463A1 (fr) * | 2013-04-11 | 2014-10-17 | Genethon | Systeme d'expression pour une therapie genique selective |
| US10292996B2 (en) * | 2015-03-26 | 2019-05-21 | The Trustees Of Columbia University In The City Of New York | Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome |
| JP6599484B2 (ja) | 2015-06-17 | 2019-10-30 | ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク | ミトコンドリアｄｎａ枯渇症候群を含む不均衡なヌクレオチドプールによって引き起こされる疾患のためのデオキシヌクレオシド療法 |
| EP3452101A2 (en) * | 2016-05-04 | 2019-03-13 | CureVac AG | Rna encoding a therapeutic protein |
-
2019
- 2019-04-18 KR KR1020207032655A patent/KR20210009317A/ko not_active Ceased
- 2019-04-18 AU AU2019255745A patent/AU2019255745A1/en not_active Abandoned
- 2019-04-18 US US17/048,236 patent/US12419970B2/en active Active
- 2019-04-18 EP EP19787753.3A patent/EP3781671A4/en active Pending
- 2019-04-18 WO PCT/US2019/028108 patent/WO2019204593A1/en not_active Ceased
- 2019-04-18 JP JP2020557222A patent/JP7769355B2/ja active Active
- 2019-04-18 BR BR112020021324-1A patent/BR112020021324A2/pt unknown
- 2019-04-18 CA CA3097392A patent/CA3097392A1/en active Pending
- 2019-04-18 MX MX2020010994A patent/MX2020010994A/es unknown
- 2019-04-18 CN CN201980039834.4A patent/CN112312929A/zh active Pending
-
2020
- 2020-10-18 IL IL278130A patent/IL278130B1/en unknown
- 2020-10-19 CL CL2020002704A patent/CL2020002704A1/es unknown
- 2020-11-17 CO CONC2020/0014193A patent/CO2020014193A2/es unknown
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