JP2025516136A5 - - Google Patents
Info
- Publication number
- JP2025516136A5 JP2025516136A5 JP2024561696A JP2024561696A JP2025516136A5 JP 2025516136 A5 JP2025516136 A5 JP 2025516136A5 JP 2024561696 A JP2024561696 A JP 2024561696A JP 2024561696 A JP2024561696 A JP 2024561696A JP 2025516136 A5 JP2025516136 A5 JP 2025516136A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- nucleic acid
- isolated nucleic
- acid molecule
- molecule 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CNPCT/CN2022/087771 | 2022-04-19 | ||
| CN2022087771 | 2022-04-19 | ||
| PCT/CN2023/089291 WO2023202637A1 (en) | 2022-04-19 | 2023-04-19 | Recombinant aav vectors for treating neurodegenerative disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2025516136A JP2025516136A (ja) | 2025-05-27 |
| JP2025516136A5 true JP2025516136A5 (https=) | 2026-04-27 |
Family
ID=88419256
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024561696A Pending JP2025516136A (ja) | 2022-04-19 | 2023-04-19 | 神経変性疾患の治療のための組み換えａａｖベクター |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US12605467B2 (https=) |
| EP (1) | EP4511482A4 (https=) |
| JP (1) | JP2025516136A (https=) |
| CN (5) | CN120866359A (https=) |
| WO (1) | WO2023202637A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN117018231B (zh) * | 2023-08-16 | 2024-05-10 | 科辉智药(深圳)新药研究中心有限公司 | 用于治疗神经病变的基因疗法及其应用 |
| WO2025166048A1 (en) * | 2024-01-31 | 2025-08-07 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Synthetic gba1 genes |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030050273A1 (en) * | 2001-08-29 | 2003-03-13 | Keiya Ozawa | Compositions and methods for treating neurodegenerative diseases |
| WO2012057363A1 (ja) * | 2010-10-27 | 2012-05-03 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| GB201118636D0 (en) * | 2011-10-28 | 2011-12-07 | Oxford Biomedica Ltd | Nucleotide sequence |
| LT3137497T (lt) * | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| RU2716991C2 (ru) * | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| EP3294890A4 (en) * | 2015-05-08 | 2018-10-03 | Children's Medical Research Institute | Promoters for expression of heterologous genes |
| KR20210059796A (ko) * | 2016-04-29 | 2021-05-25 | 오르파짐 에이/에스 | 글루코세레브로시다제 연관 장애를 치료하기 위한 아리모클로몰 |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| BR112020006661A2 (pt) * | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| WO2019068854A1 (en) * | 2017-10-06 | 2019-04-11 | Ospedale San Raffaele S.R.L. | GENE THERAPY OF NEURODEGENERATIVE DISEASES USING VAA VECTORS |
| BR112020008033A2 (pt) * | 2017-10-23 | 2020-10-27 | Prevail Therapeutics, Inc. | terapias gênicas para doença neurodegenerativa |
| CN116024271A (zh) * | 2018-05-31 | 2023-04-28 | 康霖生物科技（杭州）有限公司 | 一种用于中枢神经系统疾病治疗的基因序列构建体 |
| CN109266648B (zh) * | 2018-09-26 | 2021-10-19 | 中国科学技术大学 | 用于在体基因治疗的基因编辑组合物或试剂盒 |
| KR20220022107A (ko) * | 2018-12-05 | 2022-02-24 | 아베오나 테라퓨틱스 인코퍼레이티드 | 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 |
| CA3136004A1 (en) * | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| JP7664848B2 (ja) * | 2019-04-10 | 2025-04-18 | プリベイル セラピューティクス，インコーポレーテッド | リソソーム障害のための遺伝子療法 |
| US20220298528A1 (en) * | 2019-08-12 | 2022-09-22 | UCB Biopharma SRL | Viral particles for use in treating synucleinopathies such as parkinson's diseases by gene therapy |
| US20230047254A1 (en) * | 2019-12-29 | 2023-02-16 | Gloriana Therapeutics | Mammalian Cells Secreting GDNF and Their Therapeutic Use |
| 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 |
| WO2022032153A1 (en) * | 2020-08-06 | 2022-02-10 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| TW202325850A (zh) * | 2021-11-29 | 2023-07-01 | 大陸商上海瑞宏迪醫藥有限公司 | Aadc、gdnf多核苷酸及其用於治療帕金森病 |
| CN114317610B (zh) * | 2022-01-27 | 2024-07-02 | 上海本导基因技术有限公司 | 一种适用于帕金森疾病基因治疗的慢病毒载体 |
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2023
- 2023-04-19 EP EP23791291.0A patent/EP4511482A4/en active Pending
- 2023-04-19 JP JP2024561696A patent/JP2025516136A/ja active Pending
- 2023-04-19 CN CN202511044894.9A patent/CN120866359A/zh active Pending
- 2023-04-19 CN CN202380034877.XA patent/CN119487188A/zh active Pending
- 2023-04-19 CN CN202511044852.5A patent/CN120866357A/zh active Pending
- 2023-04-19 CN CN202511079772.3A patent/CN120866360B/zh active Active
- 2023-04-19 WO PCT/CN2023/089291 patent/WO2023202637A1/en not_active Ceased
- 2023-04-19 CN CN202511079819.6A patent/CN120866361B/zh active Active
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2024
- 2024-10-18 US US18/919,740 patent/US12605467B2/en active Active