JPWO2019173756A5 - - Google Patents

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Publication number
JPWO2019173756A5
JPWO2019173756A5 JP2020571336A JP2020571336A JPWO2019173756A5 JP WO2019173756 A5 JPWO2019173756 A5 JP WO2019173756A5 JP 2020571336 A JP2020571336 A JP 2020571336A JP 2020571336 A JP2020571336 A JP 2020571336A JP WO2019173756 A5 JPWO2019173756 A5 JP WO2019173756A5
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JP
Japan
Prior art keywords
acid sequence
seq
gba
amino acid
composition
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
JP2020571336A
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English (en)
Japanese (ja)
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JP2021517168A (ja
JP2021517168A5 (https=
Publication date
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Priority claimed from PCT/US2019/021422 external-priority patent/WO2019173756A1/en
Publication of JP2021517168A publication Critical patent/JP2021517168A/ja
Publication of JPWO2019173756A5 publication Critical patent/JPWO2019173756A5/ja
Publication of JP2021517168A5 publication Critical patent/JP2021517168A5/ja
Priority to JP2024075599A priority Critical patent/JP2024102250A/ja
Pending legal-status Critical Current

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JP2020571336A 2018-03-09 2019-03-08 パーキンソン病を処置するための組成物及び方法 Pending JP2021517168A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024075599A JP2024102250A (ja) 2018-03-09 2024-05-08 パーキンソン病を処置するための組成物及び方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862641012P 2018-03-09 2018-03-09
US62/641,012 2018-03-09
PCT/US2019/021422 WO2019173756A1 (en) 2018-03-09 2019-03-08 Compositions and methods for treating parkinson's disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024075599A Division JP2024102250A (ja) 2018-03-09 2024-05-08 パーキンソン病を処置するための組成物及び方法

Publications (3)

Publication Number Publication Date
JP2021517168A JP2021517168A (ja) 2021-07-15
JPWO2019173756A5 true JPWO2019173756A5 (https=) 2022-03-15
JP2021517168A5 JP2021517168A5 (https=) 2022-03-15

Family

ID=67847471

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020571336A Pending JP2021517168A (ja) 2018-03-09 2019-03-08 パーキンソン病を処置するための組成物及び方法
JP2024075599A Pending JP2024102250A (ja) 2018-03-09 2024-05-08 パーキンソン病を処置するための組成物及び方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024075599A Pending JP2024102250A (ja) 2018-03-09 2024-05-08 パーキンソン病を処置するための組成物及び方法

Country Status (8)

Country Link
US (1) US20210000929A1 (https=)
EP (1) EP3762505A4 (https=)
JP (2) JP2021517168A (https=)
AU (2) AU2019231889A1 (https=)
CA (1) CA3092961A1 (https=)
IL (1) IL277182A (https=)
MA (1) MA54792A (https=)
WO (1) WO2019173756A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021097131A1 (en) * 2019-11-12 2021-05-20 Orchard Therapeutics (Europe) Limited Compositions and methods for treating or preventing crohn's disease
KR20230003492A (ko) * 2020-03-29 2023-01-06 예다 리서치 앤드 디벨럽먼트 캄파니 리미티드 고셔병 치료용 베타-글루코세레브로시다아제 변이체
EP4146797A1 (en) * 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
WO2021239308A1 (en) * 2020-05-27 2021-12-02 Universität Zürich Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia
BR112023001456A2 (pt) * 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
WO2023004366A1 (en) * 2021-07-21 2023-01-26 Aspen Neuroscience, Inc. Transposon-based modulation of gba1 and related compositions and uses thereof
AU2024213347A1 (en) * 2023-02-02 2025-08-14 Embold Therapeutics, Inc. Engineered glucocerebrosidase variants
WO2024238448A2 (en) * 2023-05-12 2024-11-21 Temple University - Of The Commonwealth System Of Higher Education Chimeric capsid recombinant adeno-associated virus with bbb penetration and microglia targeting (aav-bm)
GB202307366D0 (en) * 2023-05-17 2023-06-28 Ospedale San Raffaele Srl Vector
EP4728055A2 (en) 2023-06-13 2026-04-22 Aspen Neuroscience, Inc. Methods for differentiating dopaminergic neurons from stem cells

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69727492T2 (de) * 1996-12-05 2004-12-16 Crucell Holland B.V. Genetische modifikation der hämatopoietischen repopulation von stammzellen in primaten
CN101541172A (zh) * 2005-06-08 2009-09-23 阿米库斯治疗学公司 溶酶体酶编码基因突变相关的cns紊乱的治疗
WO2012094321A1 (en) * 2011-01-03 2012-07-12 Avm Biotechnology, Llc Personalized production of biologics and method for reprogramming somatic cells
DK3444342T3 (da) * 2012-07-11 2020-08-24 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til behandlingen af lysosomale aflejringssygdomme
HUE039334T2 (hu) * 2012-11-27 2018-12-28 Biomarin Pharm Inc Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai
EP3292206B8 (en) * 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
WO2017106363A1 (en) * 2015-12-14 2017-06-22 Parkinson's Institute Refining diagnosis and treatment of complex multi-symptom neurological disorders
GB201522243D0 (en) * 2015-12-16 2016-01-27 Ucl Business Plc Treatment
AU2018354195A1 (en) * 2017-10-23 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease

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