JP2022530824A5 - - Google Patents

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Publication number
JP2022530824A5
JP2022530824A5 JP2021564730A JP2021564730A JP2022530824A5 JP 2022530824 A5 JP2022530824 A5 JP 2022530824A5 JP 2021564730 A JP2021564730 A JP 2021564730A JP 2021564730 A JP2021564730 A JP 2021564730A JP 2022530824 A5 JP2022530824 A5 JP 2022530824A5
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JP
Japan
Prior art keywords
sequence
expression cassette
raav
composition
patient
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)
Other versions
JP2022530824A (ja
Filing date
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 JP2022530824A5 publication Critical patent/JP2022530824A5/ja
Pending legal-status Critical Current

Links

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
JP2022530824A5 true JP2022530824A5 (https=) 2023-05-08

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 Before (1)

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

Country Status (16)

Country Link
US (2) US20220193207A1 (https=)
EP (2) EP3963063A4 (https=)
JP (2) JP7585233B2 (https=)
KR (2) KR20220008280A (https=)
CN (2) CN114072515A (https=)
AU (2) AU2020266829A1 (https=)
BR (2) BR112021021720A2 (https=)
CA (2) CA3134485A1 (https=)
CL (2) CL2021002755A1 (https=)
CO (2) CO2021016200A2 (https=)
IL (2) IL287522A (https=)
MX (2) MX2021013364A (https=)
PH (2) PH12021552637A1 (https=)
SG (2) SG11202111380VA (https=)
TW (1) TW202100541A (https=)
WO (2) WO2020223356A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3635009T3 (da) 2017-06-07 2026-03-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
US12258597B2 (en) 2018-02-07 2025-03-25 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
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
KR20220077921A (ko) * 2019-10-10 2022-06-09 아미쿠스 세라퓨틱스, 인코포레이티드 변이체 igf2 작제물
WO2021231863A1 (en) * 2020-05-14 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of pompe disease
AU2021292200A1 (en) 2020-06-17 2023-02-02 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
KR20240133693A (ko) * 2021-11-12 2024-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 점액다당류증 iiia의 치료를 위한 유전자 요법
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)

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Publication number Priority date Publication date Assignee Title
ES2371913T3 (es) * 2003-01-22 2012-01-11 Duke University Constructos mejorados para expresar polipéptidos lisosomales.
CA2553955C (en) * 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
EP2099523A2 (en) * 2006-11-13 2009-09-16 ZyStor Therapeutics , Inc. Methods for treating pompe disease
EP2623599B1 (en) * 2007-10-04 2019-01-02 Roche Innovation Center Copenhagen A/S Micromirs
JP5627571B2 (ja) * 2008-05-07 2014-11-19 バイオマリン ファーマシューティカル インコーポレイテッド リソソーム標的化ペプチドおよびその使用
WO2010096369A1 (en) * 2009-02-18 2010-08-26 Amicus Therapeutics, Inc. Mouse model for pompe disease and methods of use thereof
EP2401406A4 (en) * 2009-02-25 2012-09-19 Cepheid METHODS OF DETECTING LUNG CANCER
EP3679942A1 (en) * 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
ES2829199T3 (es) * 2010-11-22 2021-05-31 Amicus Therapeutics Inc Nuevas secuencias de señalización para mejorar la expresión y segregación de proteínas de enzimas recombinantes y otras proteínas
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
HRP20180291T1 (hr) * 2011-05-27 2018-03-23 Amicus Therapeutics, Inc. Postupci za sparivanje ciljajućeg peptida na rekombinantne lizosomalne enzime za poboljšano liječenje lizosomskih bolesti nakupljanja
WO2013013019A2 (en) * 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Lysosomal polypeptides, methods of making and using
US20170037431A1 (en) * 2014-05-01 2017-02-09 University Of Washington In vivo Gene Engineering with Adenoviral Vectors
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
AU2017239641A1 (en) * 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
WO2018046774A1 (en) * 2016-09-12 2018-03-15 Genethon Acid-alpha glucosidase variants and uses thereof
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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