JP2024527313A5 - - Google Patents

Info

Publication number
JP2024527313A5
JP2024527313A5 JP2023580435A JP2023580435A JP2024527313A5 JP 2024527313 A5 JP2024527313 A5 JP 2024527313A5 JP 2023580435 A JP2023580435 A JP 2023580435A JP 2023580435 A JP2023580435 A JP 2023580435A JP 2024527313 A5 JP2024527313 A5 JP 2024527313A5
Authority
JP
Japan
Prior art keywords
raav
vector
sequence
pharmaceutical composition
capsid protein
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
JP2023580435A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024527313A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/035091 external-priority patent/WO2023278305A1/en
Publication of JP2024527313A publication Critical patent/JP2024527313A/ja
Publication of JP2024527313A5 publication Critical patent/JP2024527313A5/ja
Pending legal-status Critical Current

Links

JP2023580435A 2021-06-28 2022-06-27 タウ低減遺伝子療法のための方法および組成物 Pending JP2024527313A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163215833P 2021-06-28 2021-06-28
US63/215,833 2021-06-28
US202263267440P 2022-02-02 2022-02-02
US63/267,440 2022-02-02
US202263342240P 2022-05-16 2022-05-16
US63/342,240 2022-05-16
PCT/US2022/035091 WO2023278305A1 (en) 2021-06-28 2022-06-27 Methods and compositions for tau reduction gene therapy

Publications (2)

Publication Number Publication Date
JP2024527313A JP2024527313A (ja) 2024-07-24
JP2024527313A5 true JP2024527313A5 (https=) 2025-06-25

Family

ID=82694082

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023580435A Pending JP2024527313A (ja) 2021-06-28 2022-06-27 タウ低減遺伝子療法のための方法および組成物

Country Status (6)

Country Link
US (1) US20240269161A1 (https=)
EP (1) EP4362986A1 (https=)
JP (1) JP2024527313A (https=)
AU (1) AU2022301044A1 (https=)
CA (1) CA3223506A1 (https=)
WO (1) WO2023278305A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025027577A1 (en) * 2023-08-02 2025-02-06 Consejo Nacional De Investigaciones Científicas Y Técnicas (Conicet) Micrornas designed against the microtubule-associated protein tau for the treatment of tauopathies
WO2025122644A1 (en) * 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
US20250197862A1 (en) * 2023-12-15 2025-06-19 Genzyme Corporation ARTIFICIAL microRNAs TARGETING TAU
US12551569B2 (en) 2024-03-31 2026-02-17 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of microtubule associated protein tau (MAPT), compositions thereof, and methods of use

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
DE10120265A1 (de) 2001-04-25 2002-11-14 Deutsches Krebsforsch AAV-Helferplasmide zur Helfervirus-freien Verpackung und Pseudotypisierung von AAV-Vektoren
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
CN112501208A (zh) * 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
WO2020210615A1 (en) * 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders

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