JP2022530833A5 - - Google Patents

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Publication number
JP2022530833A5
JP2022530833A5 JP2021565001A JP2021565001A JP2022530833A5 JP 2022530833 A5 JP2022530833 A5 JP 2022530833A5 JP 2021565001 A JP2021565001 A JP 2021565001A JP 2021565001 A JP2021565001 A JP 2021565001A JP 2022530833 A5 JP2022530833 A5 JP 2022530833A5
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JP
Japan
Prior art keywords
sequence
raav
expression cassette
optionally
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.)
Granted
Application number
JP2021565001A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022530833A (ja
JP7585233B2 (ja
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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 JP2022530833A5 publication Critical patent/JP2022530833A5/ja
Application granted granted Critical
Publication of JP7585233B2 publication Critical patent/JP7585233B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2021565001A 2019-04-30 2020-04-29 ポンペ病の治療のために有用な組成物 Active JP7585233B2 (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 (3)

Publication Number Publication Date
JP2022530833A JP2022530833A (ja) 2022-07-01
JP2022530833A5 true JP2022530833A5 (enExample) 2023-05-08
JP7585233B2 JP7585233B2 (ja) 2024-11-18

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 Active JP7585233B2 (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 (16)

Country Link
US (3) US20220193261A1 (enExample)
EP (2) EP3963063A4 (enExample)
JP (2) JP2022530824A (enExample)
KR (2) KR20220008280A (enExample)
CN (2) CN114127275A (enExample)
AU (2) AU2020266829A1 (enExample)
BR (2) BR112021021792A2 (enExample)
CA (2) CA3134485A1 (enExample)
CL (2) CL2021002754A1 (enExample)
CO (2) CO2021016200A2 (enExample)
IL (2) IL287522A (enExample)
MX (2) MX2021013365A (enExample)
PH (2) PH12021552638A1 (enExample)
SG (2) SG11202111380VA (enExample)
TW (1) TW202100541A (enExample)
WO (2) WO2020223362A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018281280B2 (en) 2017-06-07 2024-12-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
CN112040985A (zh) 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
JP2022552254A (ja) * 2019-10-10 2022-12-15 アミカス セラピューティックス インコーポレイテッド バリアントｉｇｆ２構築体
EP4149519A4 (en) * 2020-05-14 2024-06-12 The Trustees of The University of Pennsylvania COMPOSITIONS FOR THE TREATMENT OF POMPE DISEASE
CA3183153A1 (en) 2020-06-17 2021-12-23 Christian HINDERER Compositions and methods for treatment of gene therapy patients
AU2022388800A1 (en) * 2021-11-12 2024-06-27 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
US20260216373A1 (en) * 2023-01-13 2026-07-30 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
WO2025171163A1 (en) * 2024-02-06 2025-08-14 Duke University Compositions for and methods of treating lysosomal storage diseases

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT1587923E (pt) * 2003-01-22 2011-12-07 Univ Duke Construções melhoradas para a expressão de polipéptidos lisossomais
DE602005020745D1 (de) * 2004-02-10 2010-06-02 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
DK2205737T3 (da) * 2007-10-04 2013-05-21 Santaris Pharma As Mikromirer
WO2009137721A2 (en) * 2008-05-07 2009-11-12 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
WO2010096369A1 (en) * 2009-02-18 2010-08-26 Amicus Therapeutics, Inc. Mouse model for pompe disease and methods of use thereof
EP2628803A3 (en) 2009-02-25 2014-01-01 Cepheid Methods of detecting lung cancer
US8785168B2 (en) * 2009-06-17 2014-07-22 Biomarin Pharmaceutical Inc. Formulations for lysosomal enzymes
HRP20181315T1 (hr) 2010-11-22 2018-10-19 Amicus Therapeutics, Inc. Nove signalne sekvence za poboljšanje ekspresije proteina i izlučivanja rekombinantnih enzima i drugih proteina
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
GB201103062D0 (en) * 2011-02-22 2011-04-06 Isis Innovation Method
LT2714752T (lt) * 2011-05-27 2018-03-12 Amicus Therapeutics, Inc. Nukreipiančių peptidų kopuliavimo ant rekombinantinių lizosominių fermentų būdai, skirti lizosominio kaupimo ligų gydymo pagerinimui
WO2013013019A2 (en) * 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Lysosomal polypeptides, methods of making and using
CA2947466A1 (en) * 2014-05-01 2015-11-05 University Of Washington In vivo gene engineering with adenoviral vectors
AU2016366549B2 (en) * 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
KR102618519B1 (ko) * 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
KR102617502B1 (ko) * 2016-09-12 2023-12-26 제네똥 산-알파 글루코시다제 변이체 및 이의 용도
EP3293259A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
WO2018178067A1 (en) * 2017-03-27 2018-10-04 Vrije Universiteit Brussel Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
JP2018198611A (ja) 2018-08-03 2018-12-20 ウニベルシダッド アウトノマ デ バルセロナ 脂肪組織に形質導入するのに有用なアデノ随伴ウイルスベクター

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