JP2019511570A5 - - Google Patents

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Publication number
JP2019511570A5
JP2019511570A5 JP2018565253A JP2018565253A JP2019511570A5 JP 2019511570 A5 JP2019511570 A5 JP 2019511570A5 JP 2018565253 A JP2018565253 A JP 2018565253A JP 2018565253 A JP2018565253 A JP 2018565253A JP 2019511570 A5 JP2019511570 A5 JP 2019511570A5
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JP
Japan
Prior art keywords
composition
composition according
aav
ventricle
progranulin
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.)
Granted
Application number
JP2018565253A
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English (en)
Japanese (ja)
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JP2019511570A (ja
JP7436089B2 (ja
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Priority claimed from PCT/US2017/020397 external-priority patent/WO2017151884A1/en
Publication of JP2019511570A publication Critical patent/JP2019511570A/ja
Publication of JP2019511570A5 publication Critical patent/JP2019511570A5/ja
Priority to JP2022030769A priority Critical patent/JP7507808B2/ja
Application granted granted Critical
Publication of JP7436089B2 publication Critical patent/JP7436089B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2018565253A 2016-03-02 2017-03-02 前頭側頭型認知症の治療 Active JP7436089B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022030769A JP7507808B2 (ja) 2016-03-02 2022-03-01 前頭側頭型認知症の治療

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662302525P 2016-03-02 2016-03-02
US62/302,525 2016-03-02
PCT/US2017/020397 WO2017151884A1 (en) 2016-03-02 2017-03-02 Therapy for frontotemporal dementia

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022030769A Division JP7507808B2 (ja) 2016-03-02 2022-03-01 前頭側頭型認知症の治療

Publications (3)

Publication Number Publication Date
JP2019511570A JP2019511570A (ja) 2019-04-25
JP2019511570A5 true JP2019511570A5 (enExample) 2020-03-19
JP7436089B2 JP7436089B2 (ja) 2024-02-21

Family

ID=59743281

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018565253A Active JP7436089B2 (ja) 2016-03-02 2017-03-02 前頭側頭型認知症の治療
JP2022030769A Active JP7507808B2 (ja) 2016-03-02 2022-03-01 前頭側頭型認知症の治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022030769A Active JP7507808B2 (ja) 2016-03-02 2022-03-01 前頭側頭型認知症の治療

Country Status (6)

Country Link
US (1) US20190328906A1 (enExample)
EP (1) EP3423109A4 (enExample)
JP (2) JP7436089B2 (enExample)
AU (1) AU2017227803B2 (enExample)
CA (1) CA3016314A1 (enExample)
WO (1) WO2017151884A1 (enExample)

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CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
EP3423109A4 (en) * 2016-03-02 2019-08-14 The Children's Hospital of Philadelphia THERAPY FOR FRONTOTEMPORAL DEMENTIA
US20240002459A1 (en) * 2016-07-14 2024-01-04 Emory University Granulin Compositions and Uses Related Thereto
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
IL278868B2 (en) 2017-10-03 2025-06-01 Prevail Therapeutics Inc Gene therapies for lysosomal disorders
WO2019070891A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISORDERS
KR102700963B1 (ko) * 2017-10-03 2024-09-02 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
EP3701030A4 (en) * 2017-10-23 2022-04-20 Prevail Therapeutics, Inc. GENE THERAPIES FOR NEURODEGENERATIVE DISEASES
SG11202004926WA (en) * 2017-12-01 2020-06-29 Encoded Therapeutics Inc Engineered dna binding proteins
AU2019268330A1 (en) 2018-05-15 2020-11-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN113164627B (zh) * 2018-10-16 2024-10-29 戴纳立制药公司 治疗和监测颗粒体蛋白前体相关病症的方法
US20220111005A1 (en) 2019-02-01 2022-04-14 Avrobio, Inc. Compositions and methods for treating neurocognitive disorders
BR112021015817A2 (pt) * 2019-02-22 2021-10-13 The Trustees Of The University Of Pennsylvania Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn
MX2021012184A (es) 2019-04-10 2022-01-24 Prevail Therapeutics Inc Terapias genicas para trastornos lisosomales.
US11999974B2 (en) 2019-04-10 2024-06-04 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
BR112021024055A2 (pt) 2019-05-29 2022-02-08 Encoded Therapeutics Inc Composições e métodos para a regulação seletiva de gene
GB201913974D0 (en) 2019-09-27 2019-11-13 King S College London Vector
IL292382A (en) * 2019-10-22 2022-06-01 Applied Genetic Tech Corporation Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders
WO2021224633A1 (en) * 2020-05-06 2021-11-11 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
AU2021280943A1 (en) * 2020-05-27 2022-07-28 Universität Zürich Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia
CN116437968A (zh) * 2020-08-10 2023-07-14 普利维尔治疗公司 用于神经退行性病症的基因疗法
BR112023002374A2 (pt) * 2020-08-12 2023-03-21 UCB Biopharma SRL Terapia genética usando construções de ácido nucléico compreendendo sequências promotoras de proteína 2 de ligação de metil cpg (mecp2)
IL300622A (en) * 2020-08-26 2023-04-01 Univ Pennsylvania Recombinant adeno-associated virus for the treatment of adult-onset GRN-related neurodegeneration
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2024035649A1 (en) * 2022-08-08 2024-02-15 Shape Therapeutics Inc. Compositions for progranulin expression and methods of use thereof
GB202216168D0 (en) 2022-10-31 2022-12-14 UCB Biopharma SRL Route of administration

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BRPI0711965A2 (pt) * 2006-06-07 2012-01-24 Genzyme Corporation terapia de gene para esclerose lateral amiotrófica e outras enfermidades da medula espinal
WO2009089635A1 (en) * 2008-01-16 2009-07-23 Neurodyn, Inc. Treating neurodegenerative diseases with progranulin (pgrn)
SG10202109219SA (en) * 2009-05-02 2021-10-28 Genzyme Corp Gene therapy for neurodegenerative disorders
US20110203007A1 (en) * 2010-02-16 2011-08-18 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Assays of neurodegenerative disorders, including frontotemporal dementia and amyotrophic lateral sclerosis
EP3423109A4 (en) 2016-03-02 2019-08-14 The Children's Hospital of Philadelphia THERAPY FOR FRONTOTEMPORAL DEMENTIA

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