JPWO2020223356A5 - - Google Patents

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Publication number
JPWO2020223356A5
JPWO2020223356A5 JP2021565001A JP2021565001A JPWO2020223356A5 JP WO2020223356 A5 JPWO2020223356 A5 JP WO2020223356A5 JP 2021565001 A JP2021565001 A JP 2021565001A JP 2021565001 A JP2021565001 A JP 2021565001A JP WO2020223356 A5 JPWO2020223356 A5 JP WO2020223356A5
Authority
JP
Japan
Prior art keywords
seq
raav
expression cassette
sequence
optionally
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
JP2021565001A
Other languages
English (en)
Japanese (ja)
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JP2022530833A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/030484 external-priority patent/WO2020223356A1/en
Publication of JP2022530833A publication Critical patent/JP2022530833A/ja
Publication of JPWO2020223356A5 publication Critical patent/JPWO2020223356A5/ja
Pending legal-status Critical Current

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JP2021565001A 2019-04-30 2020-04-29 ポンペ病の治療のために有用な組成物 Pending JP2022530833A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962840911P 2019-04-30 2019-04-30
US62/840,911 2019-04-30
US201962913401P 2019-10-10 2019-10-10
US62/913,401 2019-10-10
PCT/US2020/030484 WO2020223356A1 (en) 2019-04-30 2020-04-29 Compositions useful for treatment of pompe disease

Publications (2)

Publication Number Publication Date
JP2022530833A JP2022530833A (ja) 2022-07-01
JPWO2020223356A5 true JPWO2020223356A5 (de) 2023-05-08

Family

ID=73029176

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021564730A Pending JP2022530824A (ja) 2019-04-30 2020-04-29 ポンペ病の治療のために有用な組成物
JP2021565001A Pending JP2022530833A (ja) 2019-04-30 2020-04-29 ポンペ病の治療のために有用な組成物

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2021564730A Pending JP2022530824A (ja) 2019-04-30 2020-04-29 ポンペ病の治療のために有用な組成物

Country Status (15)

Country Link
US (2) US20220193207A1 (de)
EP (2) EP3980548A4 (de)
JP (2) JP2022530824A (de)
KR (2) KR20220004696A (de)
CN (2) CN114072515A (de)
AU (2) AU2020266829A1 (de)
BR (2) BR112021021792A2 (de)
CA (2) CA3134523A1 (de)
CL (2) CL2021002755A1 (de)
CO (2) CO2021016200A2 (de)
IL (2) IL287523A (de)
MX (2) MX2021013365A (de)
SG (2) SG11202111400TA (de)
TW (1) TW202100541A (de)
WO (2) WO2020223362A1 (de)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3177954A1 (en) * 2020-05-14 2021-11-18 James M. Wilson Compositions useful for treatment of pompe disease
JP2023531451A (ja) 2020-06-17 2023-07-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 遺伝子療法患者の治療のための組成物及び方法
IL312676A (en) * 2021-11-12 2024-07-01 Amicus Therapeutics Inc Gene therapy for the treatment of mucopolysaccharidosis IIIA
NL2031676B1 (en) * 2022-04-22 2023-11-07 Univ Erasmus Med Ct Rotterdam Gene therapy for Pompe Disease
WO2024003687A1 (en) * 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
WO2024151982A1 (en) * 2023-01-13 2024-07-18 Amicus Therapeutics, Inc. Gene therapy constructs for the treatment of pompe disease
CN116693633B (zh) * 2023-02-21 2023-12-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5433133B2 (ja) * 2003-01-22 2014-03-05 デューク・ユニヴァーシティ リソソームポリペプチドを発現するための改善された構築物
ATE465250T1 (de) * 2004-02-10 2010-05-15 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
AU2007322123A1 (en) * 2006-11-13 2008-05-29 Biomarin Pharmaceutical Inc. Methods for treating Pompe disease
MY156951A (en) * 2007-10-04 2016-04-15 Santaris Pharma As Micromirs
US8466340B2 (en) * 2009-02-18 2013-06-18 Amicus Therapeutics, Inc. Mouse model for pompe disease and methods of use thereof
WO2012070014A2 (en) * 2010-11-26 2012-05-31 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Identification of novel cell surface markers for pancreatic progenitor cells and definite endodermal cells
CN108586621A (zh) * 2011-05-27 2018-09-28 阿米库斯治疗学公司 将靶向肽偶联于重组溶酶体酶上的方法
WO2013013019A2 (en) * 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Lysosomal polypeptides, methods of making and using
WO2017100671A1 (en) * 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
EP3293259A1 (de) * 2016-09-12 2018-03-14 Genethon Saure alpha-glucosidase-varianten und verwendungen davon
KR20240001721A (ko) * 2016-09-12 2024-01-03 제네똥 산-알파 글루코시다제 변이체 및 이의 용도
EP3293260A1 (de) * 2016-09-12 2018-03-14 Genethon Saure alpha-glucosidase-varianten und verwendungen davon
EP3600445A1 (de) * 2017-03-27 2020-02-05 Vrije Universiteit Brussel Membranspezifische regulatorische nukleinsäureelemente und verfahren und verwendung davon

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