JP2022554141A5 - - Google Patents
Info
- Publication number
- JP2022554141A5 JP2022554141A5 JP2022523856A JP2022523856A JP2022554141A5 JP 2022554141 A5 JP2022554141 A5 JP 2022554141A5 JP 2022523856 A JP2022523856 A JP 2022523856A JP 2022523856 A JP2022523856 A JP 2022523856A JP 2022554141 A5 JP2022554141 A5 JP 2022554141A5
- Authority
- JP
- Japan
- Prior art keywords
- patient
- aav vector
- promoter
- gaa
- aav
- 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
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962926282P | 2019-10-25 | 2019-10-25 | |
| US62/926,282 | 2019-10-25 | ||
| US202063083349P | 2020-09-25 | 2020-09-25 | |
| US63/083,349 | 2020-09-25 | ||
| PCT/US2020/057081 WO2021081338A1 (en) | 2019-10-25 | 2020-10-23 | Compositions and methods for treating glycogen storage disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022554141A JP2022554141A (ja) | 2022-12-28 |
| JP2022554141A5 true JP2022554141A5 (https=) | 2023-10-31 |
Family
ID=75620302
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022523856A Pending JP2022554141A (ja) | 2019-10-25 | 2020-10-23 | 糖原病を治療するための組成物及び方法 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20220387562A1 (https=) |
| EP (1) | EP4048286A4 (https=) |
| JP (1) | JP2022554141A (https=) |
| KR (1) | KR20220105643A (https=) |
| CN (1) | CN114828858A (https=) |
| AU (1) | AU2020372429A1 (https=) |
| BR (1) | BR112022007674A2 (https=) |
| CA (1) | CA3158281A1 (https=) |
| CO (1) | CO2022006772A2 (https=) |
| IL (1) | IL292401A (https=) |
| MX (1) | MX2022004799A (https=) |
| PH (1) | PH12022550977A1 (https=) |
| TW (2) | TW202600177A (https=) |
| WO (1) | WO2021081338A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3250992A1 (en) * | 2022-02-03 | 2023-08-10 | Astellas Gene Therapies, Inc. | COMPOSITIONS AND METHODS FOR AN IMPROVED TREATMENT OF POMPE DISEASE |
| JP2025527694A (ja) | 2022-08-23 | 2025-08-22 | マストバイオ株式会社 | Ｉｌ２変異体及びそれを含むタンパク質複合体 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2330615T3 (es) * | 2000-04-28 | 2009-12-14 | Asklepios Biopharmaceutical, Inc. | Secuencias de adn que codifican para minigenes de distrofina y metodos de uso de las mismas. |
| EP1366160B1 (en) * | 2000-10-06 | 2008-07-09 | The Regents Of The University Of Michigan | Mini-dystrophin nucleic acid and peptide sequences |
| KR20150020250A (ko) * | 2012-06-19 | 2015-02-25 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 당뇨병을 치료하기 위한 조성물 및 방법 |
| IL258005B2 (en) * | 2015-09-17 | 2025-01-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2 |
| EP3293260A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3596112A2 (en) * | 2017-03-17 | 2020-01-22 | Newcastle University | Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy |
| US11597917B2 (en) * | 2017-07-06 | 2023-03-07 | The Medical College Of Wisconsin, Inc. | In vitro and in vivo enrichment strategy targeting lymphocytes derived from vector transduced HSCs for therapy of disorders |
| CN111936621B (zh) * | 2018-02-05 | 2025-05-13 | 奥登茨治疗公司 | 转录调控元件和它的用途 |
| CN111902539B (zh) * | 2018-02-07 | 2025-05-16 | 吉尼松公司 | 杂合调控元件 |
-
2020
- 2020-10-23 JP JP2022523856A patent/JP2022554141A/ja active Pending
- 2020-10-23 CA CA3158281A patent/CA3158281A1/en active Pending
- 2020-10-23 IL IL292401A patent/IL292401A/en unknown
- 2020-10-23 TW TW114116784A patent/TW202600177A/zh unknown
- 2020-10-23 EP EP20880074.8A patent/EP4048286A4/en active Pending
- 2020-10-23 CN CN202080086069.4A patent/CN114828858A/zh active Pending
- 2020-10-23 TW TW109136852A patent/TWI885000B/zh active
- 2020-10-23 AU AU2020372429A patent/AU2020372429A1/en active Pending
- 2020-10-23 US US17/771,627 patent/US20220387562A1/en active Pending
- 2020-10-23 MX MX2022004799A patent/MX2022004799A/es unknown
- 2020-10-23 BR BR112022007674A patent/BR112022007674A2/pt unknown
- 2020-10-23 WO PCT/US2020/057081 patent/WO2021081338A1/en not_active Ceased
- 2020-10-23 PH PH1/2022/550977A patent/PH12022550977A1/en unknown
- 2020-10-23 KR KR1020227017631A patent/KR20220105643A/ko active Pending
-
2022
- 2022-05-23 CO CONC2022/0006772A patent/CO2022006772A2/es unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20220331409A1 (en) | Factor ix gene therapy | |
| US20250161490A1 (en) | Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy | |
| JP2021512649A5 (https=) | ||
| CN102227500A (zh) | 胆色素原脱氨酶基因治疗 | |
| JP2020513811A5 (https=) | ||
| JP2021522173A5 (https=) | ||
| US11891616B2 (en) | Transgene cassettes designed to express a human MECP2 gene | |
| EP3495472B1 (en) | Recombinant virus products and methods for inhibition of expression of myotilin | |
| WO2021034222A9 (ru) | Выделенный модифицированный белок vp1 капсида aav5 | |
| CA3248774A1 (en) | ANTHANOGENE 3 ASSOCIATED WITH B-LYMPHOMA 2 INVOLVING AN ADENO-ASSOCIATED VIRUS (AAV) VECTOR | |
| JP2023537358A5 (https=) | ||
| CN114466682A (zh) | 用于治疗基于肌营养不良蛋白的肌病的靶向外显子44的核酸和包含所述核酸的重组腺相关病毒 | |
| EP4536686A1 (en) | Peptide-modified aav capsid with enhanced muscle transduction efficiency | |
| JP2022554141A5 (https=) | ||
| JP2023553005A (ja) | 末梢ミエリンタンパク質２２の発現の阻害のための生成物及び方法 | |
| JP2025509262A5 (https=) | ||
| Flotte | Adeno-associated virus-mediated gene transfer for lung diseases | |
| JP2022523320A5 (https=) | ||
| JP2025505575A5 (https=) | ||
| WO2025162449A1 (en) | Compositions and methods for the treatment of heart disease | |
| CA3236188A1 (en) | Aav-idua vector for treatment of mps i | |
| TW202434729A (zh) | 含有星狀細胞導向啟動子構築體之合成核酸及其使用方法 | |
| HK40118571A (zh) | 腺相关病毒载体传递微肌营养不良蛋白以治疗肌营养不良症 | |
| RU2024125625A (ru) | Композиции и способы для оптимизированного лечения болезни помпе | |
| JP2021527418A (ja) | ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法 |