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 (es) 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 After (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 (es)
EP (2) EP3963063A4 (es)
JP (2) JP2022530824A (es)
KR (2) KR20220008280A (es)
CN (2) CN114127275A (es)
AU (2) AU2020266552A1 (es)
BR (2) BR112021021792A2 (es)
CA (2) CA3134523A1 (es)
CL (2) CL2021002755A1 (es)
CO (2) CO2021016200A2 (es)
IL (2) IL287522A (es)
MX (2) MX2021013364A (es)
SG (2) SG11202111380VA (es)
TW (1) TW202100541A (es)
WO (2) WO2020223356A1 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023526923A (ja) * 2020-05-14 2023-06-26 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 ポンペ病の治療に有用な組成物
AU2021292200A1 (en) 2020-06-17 2023-02-02 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
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
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
ES2371913T3 (es) * 2003-01-22 2012-01-11 Duke University Constructos mejorados para expresar polipéptidos lisosomales.
DE602005020745D1 (de) * 2004-02-10 2010-06-02 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
EP2099523A2 (en) * 2006-11-13 2009-09-16 ZyStor Therapeutics , Inc. Methods for treating pompe disease
US8440637B2 (en) * 2007-10-04 2013-05-14 Santaris Pharma A/S Combination treatment for the treatment of hepatitis C virus infection
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
KR101955054B1 (ko) * 2011-05-27 2019-03-07 아미쿠스 세라퓨틱스, 인코포레이티드 리소좀 축적증의 개선된 치료를 위해 재조합 리소좀 효소상에 표적화 펩티드를 커플링시키는 방법
WO2013013019A2 (en) * 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Lysosomal polypeptides, methods of making and using
AU2016366549B2 (en) * 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
MX2019002842A (es) * 2016-09-12 2019-08-29 Genethon Variantes de alfa-glucosidasa acida y usos de las mismas.
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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