JPWO2020223362A5 - - Google Patents

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Publication number
JPWO2020223362A5
JPWO2020223362A5 JP2021564730A JP2021564730A JPWO2020223362A5 JP WO2020223362 A5 JPWO2020223362 A5 JP WO2020223362A5 JP 2021564730 A JP2021564730 A JP 2021564730A JP 2021564730 A JP2021564730 A JP 2021564730A JP WO2020223362 A5 JPWO2020223362 A5 JP WO2020223362A5
Authority
JP
Japan
Prior art keywords
raav
expression cassette
composition
sequence
seq
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
JP2021564730A
Other languages
English (en)
Japanese (ja)
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JP2022530824A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/030493 external-priority patent/WO2020223362A1/en
Publication of JP2022530824A publication Critical patent/JP2022530824A/ja
Publication of JPWO2020223362A5 publication Critical patent/JPWO2020223362A5/ja
Pending legal-status Critical Current

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JP2021564730A 2019-04-30 2020-04-29 ポンペ病の治療のために有用な組成物 Pending JP2022530824A (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/US2020/030493 WO2020223362A1 (en) 2019-04-30 2020-04-29 Compositions useful for treatment of pompe disease

Publications (2)

Publication Number Publication Date
JP2022530824A JP2022530824A (ja) 2022-07-01
JPWO2020223362A5 true JPWO2020223362A5 (he) 2023-05-08

Family

ID=73029176

Family Applications (2)

Application Number Title Priority Date Filing Date
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
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

Family Applications Before (1)

Application Number Title Priority Date Filing Date
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

Country Status (15)

Country Link
US (2) US20220193261A1 (he)
EP (2) EP3963063A4 (he)
JP (2) JP2022530833A (he)
KR (2) KR20220008280A (he)
CN (2) CN114072515A (he)
AU (2) AU2020266552A1 (he)
BR (2) BR112021021720A2 (he)
CA (2) CA3134523A1 (he)
CL (2) CL2021002754A1 (he)
CO (2) CO2021016200A2 (he)
IL (2) IL287522A (he)
MX (2) MX2021013365A (he)
SG (2) SG11202111380VA (he)
TW (1) TW202100541A (he)
WO (2) WO2020223356A1 (he)

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
CN115968302A (zh) 2020-06-17 2023-04-14 宾夕法尼亚州大学信托人 用于治疗基因疗法患者的组合物和方法
WO2023086928A2 (en) * 2021-11-12 2023-05-19 The Trustees Of The University Of Pennsylvania Gene therapy for 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
US20040248262A1 (en) * 2003-01-22 2004-12-09 Koeberl Dwight D. Constructs for expressing lysomal polypeptides
US20050244400A1 (en) * 2004-02-10 2005-11-03 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
WO2008063511A2 (en) * 2006-11-13 2008-05-29 Zystor Therapeutics, Inc. Methods for treating pompe disease
CN101821391B (zh) * 2007-10-04 2016-04-27 桑塔里斯制药公司 微小聚体
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
WO2012166653A2 (en) * 2011-05-27 2012-12-06 Callidus Biopharma, Inc. Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases
WO2013013019A2 (en) * 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Lysosomal polypeptides, methods of making and using
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
KR20240001721A (ko) * 2016-09-12 2024-01-03 제네똥 산-알파 글루코시다제 변이체 및 이의 용도
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293259A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
JP2020515572A (ja) * 2017-03-27 2020-05-28 ブレイエ・ユニバージテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｔｅｉｔ Ｂｒｕｓｓｅｌ 横隔膜特異的核酸調節エレメントならびにその方法および使用

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