JP2018537984A5 - - Google Patents
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- JP2018537984A5 JP2018537984A5 JP2018531163A JP2018531163A JP2018537984A5 JP 2018537984 A5 JP2018537984 A5 JP 2018537984A5 JP 2018531163 A JP2018531163 A JP 2018531163A JP 2018531163 A JP2018531163 A JP 2018531163A JP 2018537984 A5 JP2018537984 A5 JP 2018537984A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- aav
- composition
- sequence
- vector according
- 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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Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267012P | 2015-12-14 | 2015-12-14 | |
| US62/267,012 | 2015-12-14 | ||
| PCT/US2016/066669 WO2017106354A1 (en) | 2015-12-14 | 2016-12-14 | Adeno-associated viral vectors useful in treatment of spinal muscular atropy |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018537984A JP2018537984A (ja) | 2018-12-27 |
| JP2018537984A5 true JP2018537984A5 (enExample) | 2020-01-23 |
| JP7082050B2 JP7082050B2 (ja) | 2022-06-07 |
Family
ID=57737990
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018531163A Expired - Fee Related JP7082050B2 (ja) | 2015-12-14 | 2016-12-14 | 脊髄性筋萎縮症の処置において有用なアデノ－関連ウイルスベクター |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US20180353624A1 (enExample) |
| EP (1) | EP3394270A1 (enExample) |
| JP (1) | JP7082050B2 (enExample) |
| KR (1) | KR20180086266A (enExample) |
| CN (1) | CN109072254A (enExample) |
| AU (1) | AU2016370630B2 (enExample) |
| BR (1) | BR112018011975A2 (enExample) |
| CA (1) | CA3008280A1 (enExample) |
| IL (1) | IL259877A (enExample) |
| MA (1) | MA44119A (enExample) |
| MX (1) | MX2018007234A (enExample) |
| WO (1) | WO2017106354A1 (enExample) |
| ZA (1) | ZA201803956B (enExample) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102621134B1 (ko) * | 2016-07-26 | 2024-01-04 | 코넬 유니버시티 | 알데히드 데히드로게나제 결핍의 치료를 위한 유전자 요법 |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| SI3589730T1 (sl) | 2017-02-28 | 2024-04-30 | The Trustees Of The University Of Pennsylvania | Adeno-povezan virus (AAV) clade F vektor in njihova uporaba |
| WO2019040586A1 (en) * | 2017-08-25 | 2019-02-28 | Ovid Therapeutics Inc. | RECOMBINANT ADENO-ASSOCIATE VECTORS |
| US20210308281A1 (en) * | 2018-08-15 | 2021-10-07 | Biogen Ma Inc. | Combination therapy for spinal muscular atrophy |
| CN113474459A (zh) * | 2018-12-21 | 2021-10-01 | 吉尼松公司 | 用于基因疗法载体的表达盒 |
| US20220280548A1 (en) * | 2019-08-15 | 2022-09-08 | Biogen Ma Inc. | Combination therapy for spinal muscular atrophy |
| CN112011571A (zh) * | 2020-04-26 | 2020-12-01 | 辉大(上海)生物科技有限公司 | 一种用于治疗脊髓性肌萎缩的基因治疗药物 |
| US20230173102A1 (en) * | 2020-04-28 | 2023-06-08 | Genethon | Use of a Synthetic AAV Capsid for Gene Therapy of Muscle and Central Nervous System Disorders |
| EP4162059A1 (en) | 2020-05-12 | 2023-04-12 | The Trustees of The University of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| RU2742837C1 (ru) * | 2020-06-02 | 2021-02-11 | Общество С Ограниченной Ответственностью "Анабион" | Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение |
| CN113755524B (zh) * | 2020-06-02 | 2023-11-03 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途 |
| TW202246501A (zh) * | 2021-01-29 | 2022-12-01 | 俄羅斯聯邦商亞那拜恩有限公司 | ＳＭＮ１和ｍｉＲ－２３ａ在治療脊髓性肌萎縮中的協同作用 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| CN112852882A (zh) * | 2021-02-04 | 2021-05-28 | 中吉智药(南京)生物技术有限公司 | 一种杆状病毒感染昆虫细胞生产aav基因药物的系统及方法 |
| KR20240101620A9 (ko) * | 2021-11-09 | 2025-12-10 | 아시모브 인코포레이티드 | Aav 벡터 생산을 위한 안정한 생산 시스템 |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2023219394A1 (ko) * | 2022-05-10 | 2023-11-16 | 서울대학교산학협력단 | 인간 smn1 단백질 변이체 및 이의 용도 |
| WO2024165839A1 (en) * | 2023-02-06 | 2024-08-15 | Royal Holloway And Bedford New College | Transgenes |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK3211085T3 (da) * | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| WO2013190059A1 (en) * | 2012-06-21 | 2013-12-27 | Association Institut De Myologie | Widespread gene delivery of gene therapy vectors |
| US10821154B2 (en) * | 2013-05-01 | 2020-11-03 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
| CN105658800A (zh) | 2013-10-22 | 2016-06-08 | 夏尔人类遗传性治疗公司 | Mrna的cns递送及其用途 |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| CA2994160C (en) * | 2015-07-30 | 2021-08-10 | Massachusetts Eye And Ear Infirmary | Ancestral virus sequences and uses thereof |
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2016
- 2016-12-14 WO PCT/US2016/066669 patent/WO2017106354A1/en not_active Ceased
- 2016-12-14 US US16/061,109 patent/US20180353624A1/en not_active Abandoned
- 2016-12-14 EP EP16822581.1A patent/EP3394270A1/en not_active Withdrawn
- 2016-12-14 MX MX2018007234A patent/MX2018007234A/es unknown
- 2016-12-14 KR KR1020187019828A patent/KR20180086266A/ko not_active Withdrawn
- 2016-12-14 JP JP2018531163A patent/JP7082050B2/ja not_active Expired - Fee Related
- 2016-12-14 MA MA044119A patent/MA44119A/fr unknown
- 2016-12-14 BR BR112018011975A patent/BR112018011975A2/pt not_active Application Discontinuation
- 2016-12-14 CA CA3008280A patent/CA3008280A1/en active Pending
- 2016-12-14 AU AU2016370630A patent/AU2016370630B2/en not_active Expired - Fee Related
- 2016-12-14 CN CN201680081819.2A patent/CN109072254A/zh active Pending
-
2018
- 2018-06-07 IL IL259877A patent/IL259877A/en unknown
- 2018-06-13 ZA ZA2018/03956A patent/ZA201803956B/en unknown
-
2021
- 2021-09-30 US US17/490,611 patent/US20220265861A1/en not_active Abandoned