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
JP7585233B2 (ja
JP2022530833A (ja
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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 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 (OSRAM) 2023-05-08
JP7585233B2 JP7585233B2 (ja) 2024-11-18

Family

ID=73029176

Family Applications (2)

Application Number Title Priority Date Filing Date
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
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 After (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 (2) US20220193261A1 (OSRAM)
EP (2) EP3963063A4 (OSRAM)
JP (2) JP7585233B2 (OSRAM)
KR (2) KR20220008280A (OSRAM)
CN (2) CN114127275A (OSRAM)
AU (2) AU2020266829A1 (OSRAM)
BR (2) BR112021021792A2 (OSRAM)
CA (2) CA3134485A1 (OSRAM)
CL (2) CL2021002755A1 (OSRAM)
CO (2) CO2021016198A2 (OSRAM)
IL (2) IL287523A (OSRAM)
MX (2) MX2021013365A (OSRAM)
PH (2) PH12021552637A1 (OSRAM)
SG (2) SG11202111380VA (OSRAM)
TW (1) TW202100541A (OSRAM)
WO (2) WO2020223356A1 (OSRAM)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES3065816T3 (en) 2017-06-07 2026-05-08 Regeneron Pharma 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
BR112020023145A2 (pt) 2018-05-17 2021-02-02 Regeneron Pharmaceuticals, Inc. anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto
AU2020361703A1 (en) * 2019-10-10 2022-04-28 Amicus Therapeutics, Inc. Variant IGF2 constructs
MX2022014255A (es) * 2020-05-14 2022-12-07 Univ Pennsylvania Composiciones utiles para el tratamiento de la enfermedad de pompe.
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
CA3237987A1 (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
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
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
CA2553955C (en) * 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
CA2669347A1 (en) * 2006-11-13 2008-05-29 Zystor Therapeutics, Inc. Methods for treating pompe disease
ES2463665T3 (es) * 2007-10-04 2014-05-28 Stella Aps Tratamiento de combinación para el tratamiento de infección por virus de la hepatitis C
ES2629853T3 (es) * 2008-05-07 2017-08-16 Biomarin Pharmaceutical Inc. Péptidos de dirección lisosómica y usos de los mismos
US8466340B2 (en) * 2009-02-18 2013-06-18 Amicus Therapeutics, Inc. Mouse model for pompe disease and methods of use thereof
WO2010099342A2 (en) * 2009-02-25 2010-09-02 Cepheid Methods of detecting lung cancer
EP2475376B1 (en) * 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
DK2643468T3 (en) 2010-11-22 2018-09-24 Amicus Therapeutics Inc Hitherto UNKNOWN SIGNAL SEQUENCES TO IMPROVE EXPRESSION AND SECRETARY OF RECOMBINANT ENZYMES AND OTHER PROTEINS
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
TR201802230T4 (tr) * 2011-05-27 2018-03-21 Amicus Therapeutics Inc Lizozomal depo hastalıklarının gelişmiş tedavileri için hedefleyici peptitlerin rekombinan lizozomal enzimler üzerine kenetlenmesi için yöntemler.
WO2013013019A2 (en) * 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Lysosomal polypeptides, methods of making and using
JP2017514476A (ja) * 2014-05-01 2017-06-08 ユニバーシティ・オブ・ワシントン アデノウイルスベクターを用いたインビボでの遺伝子操作
EP3875593A1 (en) * 2015-12-11 2021-09-08 California Institute of Technology Targeting peptides for directing adeno-associated viruses (aavs)
SG11201808455VA (en) * 2016-03-30 2018-10-30 Amicus Therapeutics Inc Formulations comprising recombinant acid alpha-glucosidase
EP3293259A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
PE20191203A1 (es) * 2016-09-12 2019-09-10 Genethon Variantes de alfa-glucosidasa acida y usos de las mismas
EP3600445A1 (en) * 2017-03-27 2020-02-05 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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