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
Application number
JP2024561696A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025516136A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/CN2023/089291 external-priority patent/WO2023202637A1/en
Publication of JP2025516136A publication Critical patent/JP2025516136A/ja
Publication of JP2025516136A5 publication Critical patent/JP2025516136A5/ja
Pending legal-status Critical Current

Links

JP2024561696A 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 Pending JP2025516136A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2022087771 2022-04-19
CNPCT/CN2022/087771 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 (enExample) 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 (2) US12605467B2 (enExample)
EP (1) EP4511482A4 (enExample)
JP (1) JP2025516136A (enExample)
CN (5) CN120866360B (enExample)
WO (1) WO2023202637A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
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 (24)

* Cited by examiner, † Cited by third party
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
WO2006063247A2 (en) * 2004-12-09 2006-06-15 Genzyme Corporation Regulated expression of transgenes in the central nervous system of mammals
US10738326B2 (en) * 2010-10-27 2020-08-11 Jichi Medical University Adeno-associated virus vector for gene transfer to nervous system cells
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
EP3215191B1 (en) * 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
AU2016259976B2 (en) * 2015-05-08 2021-10-14 Children's Medical Research Institute Promoters for expression of heterologous genes
ES2831764T3 (es) * 2016-04-29 2021-06-09 Orphazyme As Arimoclomol para el tratamiento de trastornos asociados a glucocerebrosidasa
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
EP3692158A4 (en) * 2017-10-03 2021-08-04 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
WO2019068854A1 (en) * 2017-10-06 2019-04-11 Ospedale San Raffaele S.R.L. GENE THERAPY OF NEURODEGENERATIVE DISEASES USING VAA VECTORS
MX2020004035A (es) * 2017-10-20 2020-11-09 Res Inst Nationwide Childrens Hospital Métodos y materiales para terapia génica con nt-3.
AU2018354195A1 (en) * 2017-10-23 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
CN108841868A (zh) * 2018-05-31 2018-11-20 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
BR112021009913A2 (pt) * 2018-12-05 2021-08-17 Abeona Therapeutics, Inc. vetor viral adeno-associado recombinante para inserção gênica
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
HRP20251506T1 (hr) * 2019-04-10 2026-01-02 Prevail Therapeutics, Inc. Genska terapija za lizosomske poremećaje
CA3149844A1 (en) * 2019-08-12 2021-02-18 Fundacion Para La Investigacion Medica Aplicada 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
EP4192514A1 (en) * 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
CA3238867A1 (en) * 2021-11-29 2023-06-01 Shanghai Regenelead Therapies Co., Ltd. Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
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

Similar Documents

Publication Publication Date Title
JP2023534999A5 (enExample)
JP2022530656A5 (enExample)
JP2022530633A5 (enExample)
JP2022530544A5 (enExample)
JP2021515548A5 (enExample)
JP2025032120A5 (enExample)
JP2021516046A5 (enExample)
JP2020510430A5 (enExample)
IL273261B2 (en) Adeno-associated virus variant capsids and methods of using them
JP2022549380A5 (enExample)
JP2022531861A5 (enExample)
JP2022500066A5 (enExample)
JP2022547197A5 (enExample)
JP2023537625A5 (enExample)
JP2022523679A5 (enExample)
US5858775A (en) Adeno-associated virus materials and methods
JP2022541070A5 (enExample)
JP2025517628A5 (enExample)
JP2023535620A5 (enExample)
JP2022529782A5 (enExample)
JP2025500347A5 (enExample)
JP2024542950A5 (enExample)
JP2024530023A5 (enExample)
JP2023537358A5 (enExample)
JP2023550581A5 (enExample)