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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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 (https=) | 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 (https=) |
| EP (1) | EP3394270A1 (https=) |
| JP (1) | JP7082050B2 (https=) |
| KR (1) | KR20180086266A (https=) |
| CN (1) | CN109072254A (https=) |
| AU (1) | AU2016370630B2 (https=) |
| BR (1) | BR112018011975A2 (https=) |
| CA (1) | CA3008280A1 (https=) |
| IL (1) | IL259877A (https=) |
| MA (1) | MA44119A (https=) |
| MX (1) | MX2018007234A (https=) |
| WO (1) | WO2017106354A1 (https=) |
| ZA (1) | ZA201803956B (https=) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2017301819B2 (en) * | 2016-07-26 | 2024-09-26 | Cornell University | Gene therapy for the treatment of aldehyde dehydrogenase deficiency |
| JP7455579B2 (ja) | 2017-02-28 | 2024-03-26 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 新規アデノ随伴ウイルス(aav)クレードfベクター及びその用途 |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| MX2020002148A (es) * | 2017-08-25 | 2020-07-20 | Ovid Therapeutics Inc | Vectores adenoasociados recombinantes. |
| EP3837374A4 (en) * | 2018-08-15 | 2022-06-08 | Biogen MA Inc. | COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY |
| EP3898995A1 (en) * | 2018-12-21 | 2021-10-27 | Genethon | Expression cassettes for gene therapy vectors |
| 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 |
| US20230304034A1 (en) | 2020-05-12 | 2023-09-28 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| 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 |
| RU2742837C1 (ru) * | 2020-06-02 | 2021-02-11 | Общество С Ограниченной Ответственностью "Анабион" | Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение |
| MA62178A1 (fr) * | 2021-01-29 | 2023-12-29 | Biocad Joint Stock Co | Effet synergique de smn1 et mir-23a dans le traitement de l'amyotrophie spinale |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | 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 |
| EP4430170A2 (en) * | 2021-11-09 | 2024-09-18 | Asimov, Inc. | Stable production systems for aav vector production |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| KR102717399B1 (ko) * | 2022-05-10 | 2024-10-16 | 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 |
|---|---|---|---|---|
| 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 |
| WO2013190059A1 (en) * | 2012-06-21 | 2013-12-27 | Association Institut De Myologie | Widespread gene delivery of gene therapy vectors |
| MX380973B (es) * | 2013-05-01 | 2025-03-12 | Genzyme Corp | Composiciones y metodos para tratar la atrofia muscular espinal. |
| AU2014340149B2 (en) | 2013-10-22 | 2020-12-24 | Shire Human Genetic Therapies, Inc. | CNS delivery of mRNA and uses thereof |
| 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 BR BR112018011975A patent/BR112018011975A2/pt not_active Application Discontinuation
- 2016-12-14 MA MA044119A patent/MA44119A/fr unknown
- 2016-12-14 KR KR1020187019828A patent/KR20180086266A/ko not_active Withdrawn
- 2016-12-14 US US16/061,109 patent/US20180353624A1/en not_active Abandoned
- 2016-12-14 AU AU2016370630A patent/AU2016370630B2/en not_active Expired - Fee Related
- 2016-12-14 CA CA3008280A patent/CA3008280A1/en active Pending
- 2016-12-14 JP JP2018531163A patent/JP7082050B2/ja not_active Expired - Fee Related
- 2016-12-14 EP EP16822581.1A patent/EP3394270A1/en not_active Withdrawn
- 2016-12-14 MX MX2018007234A patent/MX2018007234A/es unknown
- 2016-12-14 CN CN201680081819.2A patent/CN109072254A/zh active Pending
- 2016-12-14 WO PCT/US2016/066669 patent/WO2017106354A1/en not_active Ceased
-
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