JP2022513034A5 - - Google Patents
Info
- Publication number
- JP2022513034A5 JP2022513034A5 JP2021526294A JP2021526294A JP2022513034A5 JP 2022513034 A5 JP2022513034 A5 JP 2022513034A5 JP 2021526294 A JP2021526294 A JP 2021526294A JP 2021526294 A JP2021526294 A JP 2021526294A JP 2022513034 A5 JP2022513034 A5 JP 2022513034A5
- Authority
- JP
- Japan
- Prior art keywords
- approximately
- pharmaceutical composition
- subject
- raav
- composition 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.)
- Pending
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025076129A JP2025128087A (ja) | 2018-11-14 | 2025-05-01 | 神経セロイドリポフスチン症の遺伝子治療 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862767410P | 2018-11-14 | 2018-11-14 | |
| US62/767,410 | 2018-11-14 | ||
| US201962924060P | 2019-10-21 | 2019-10-21 | |
| US62/924,060 | 2019-10-21 | ||
| PCT/US2019/061206 WO2020102369A1 (en) | 2018-11-14 | 2019-11-13 | Gene therapy for neuronal ceroid lipofuscinoses |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025076129A Division JP2025128087A (ja) | 2018-11-14 | 2025-05-01 | 神経セロイドリポフスチン症の遺伝子治療 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022513034A JP2022513034A (ja) | 2022-02-07 |
| JP2022513034A5 true JP2022513034A5 (https=) | 2023-01-26 |
Family
ID=70731911
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021526294A Pending JP2022513034A (ja) | 2018-11-14 | 2019-11-13 | 神経セロイドリポフスチン症の遺伝子治療 |
| JP2025076129A Pending JP2025128087A (ja) | 2018-11-14 | 2025-05-01 | 神経セロイドリポフスチン症の遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025076129A Pending JP2025128087A (ja) | 2018-11-14 | 2025-05-01 | 神経セロイドリポフスチン症の遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20210393802A1 (https=) |
| EP (1) | EP3880235A4 (https=) |
| JP (2) | JP2022513034A (https=) |
| KR (1) | KR20210092755A (https=) |
| AU (1) | AU2019379141A1 (https=) |
| BR (1) | BR112021009370A2 (https=) |
| CA (1) | CA3117982A1 (https=) |
| IL (2) | IL315701B1 (https=) |
| MX (1) | MX2021005517A (https=) |
| SG (1) | SG11202104295UA (https=) |
| WO (1) | WO2020102369A1 (https=) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| WO2020191233A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| PT4021575T (pt) * | 2019-08-29 | 2024-07-30 | Biomarin Pharm Inc | Métodos para o tratamento da doença cln2 em sujeitos pediátricos |
| WO2021071835A1 (en) * | 2019-10-07 | 2021-04-15 | Regenxbio Inc. | Adeno-associated virus vector pharmaceutical composition and methods |
| AU2021358781A1 (en) | 2020-10-07 | 2023-05-18 | Tern Therapeutics, Llc | Gene therapy for ocular manifestations of cln2 disease |
| US20240066146A1 (en) * | 2020-12-18 | 2024-02-29 | Sangamo Therapeutics, Inc. | Improved pharmaceutical compositions containing adeno-associated viral vector |
| WO2022165313A1 (en) * | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| EP4323531A1 (en) * | 2021-04-12 | 2024-02-21 | Affinia Therapeutics Inc. | Recombinant aav for treatment of neural disease |
| WO2023113806A1 (en) * | 2021-12-16 | 2023-06-22 | Affinia Therapeutics, Inc. | Recombinant aav for treatment of neural disease |
| AU2022279062A1 (en) * | 2021-05-17 | 2024-01-04 | Sarepta Therapeutics, Inc. | Production of recombinant aav vectors for treating muscular dystrophy |
| WO2023177626A1 (en) * | 2022-03-14 | 2023-09-21 | Regeneron Pharmaceuticals, Inc. | Lyophilized formulations of aav drug products |
| US20260034243A1 (en) * | 2022-04-06 | 2026-02-05 | Regenxbio Inc. | Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene for suprachoroidal administration |
| CN115869425B (zh) * | 2022-12-12 | 2024-08-20 | 北京生物制品研究所有限责任公司 | 一种aav眼用注射液及其制备方法和应用 |
| WO2025122725A1 (en) * | 2023-12-06 | 2025-06-12 | The Broad Institute, Inc. | Methods and compositions for base editing of tpp1 in the treatment of batten disease |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20120322861A1 (en) * | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| JP5332064B2 (ja) * | 2007-03-01 | 2013-11-06 | ウェルスタット イムノセラピューティクス， エルエルシー | 炎症により特徴付けられる疾患の治療 |
| WO2012135857A1 (en) * | 2011-03-31 | 2012-10-04 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| CA2853379C (en) * | 2011-10-27 | 2020-11-24 | Wellstat Ophthalmics Corporation | Vectors encoding rod-derived cone viability factor |
| CA2853482C (en) * | 2011-10-28 | 2020-05-19 | Joshua Grieger | Cell line for production of adeno-associated virus |
| US20140314740A1 (en) * | 2011-12-09 | 2014-10-23 | The Johns Hopkins University | Compositions and methods for the prevention or treatment of diabetic complications |
| US20140359799A1 (en) * | 2011-12-23 | 2014-12-04 | Case Western Reserve University | Targeted gene modification using hybrid recombinant adeno-associated virus |
| CN105745326A (zh) * | 2013-10-24 | 2016-07-06 | 优尼科Ip有限公司 | 用于基因治疗神经疾病的aav-5假型载体 |
| CN107530447A (zh) * | 2015-02-10 | 2018-01-02 | 建新公司 | 病毒颗粒至纹状体和皮质的增强递送 |
| TWI752907B (zh) * | 2015-05-08 | 2022-01-21 | 美商拜奧馬林製藥公司 | 用於治療cln2疾病之tpp1調配物及方法 |
| SG10201912761UA (en) * | 2016-04-15 | 2020-02-27 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| JP7610923B2 (ja) * | 2016-09-02 | 2025-01-09 | スパーク セラピューティクス インコーポレイテッド | Ｃｎｓ障害を治療するための方法及びベクター |
| CA3041548A1 (en) * | 2016-11-04 | 2018-05-11 | The Children's Hospital Of Philadelphia | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
| JOP20190200A1 (ar) * | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| IL316926A (en) * | 2017-05-11 | 2025-01-01 | Univ Pennsylvania | Gene therapy for neuronal ceroid lipofuscinosis |
-
2019
- 2019-11-13 BR BR112021009370-2A patent/BR112021009370A2/pt not_active IP Right Cessation
- 2019-11-13 CA CA3117982A patent/CA3117982A1/en active Pending
- 2019-11-13 JP JP2021526294A patent/JP2022513034A/ja active Pending
- 2019-11-13 IL IL315701A patent/IL315701B1/en unknown
- 2019-11-13 KR KR1020217017295A patent/KR20210092755A/ko not_active Ceased
- 2019-11-13 WO PCT/US2019/061206 patent/WO2020102369A1/en not_active Ceased
- 2019-11-13 IL IL282808A patent/IL282808B2/en unknown
- 2019-11-13 AU AU2019379141A patent/AU2019379141A1/en not_active Abandoned
- 2019-11-13 SG SG11202104295UA patent/SG11202104295UA/en unknown
- 2019-11-13 US US17/292,750 patent/US20210393802A1/en active Pending
- 2019-11-13 MX MX2021005517A patent/MX2021005517A/es unknown
- 2019-11-13 EP EP19884046.4A patent/EP3880235A4/en active Pending
-
2025
- 2025-05-01 JP JP2025076129A patent/JP2025128087A/ja active Pending
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