JPWO2021081338A5 - - Google Patents

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Publication number
JPWO2021081338A5
JPWO2021081338A5 JP2022523856A JP2022523856A JPWO2021081338A5 JP WO2021081338 A5 JPWO2021081338 A5 JP WO2021081338A5 JP 2022523856 A JP2022523856 A JP 2022523856A JP 2022523856 A JP2022523856 A JP 2022523856A JP WO2021081338 A5 JPWO2021081338 A5 JP WO2021081338A5
Authority
JP
Japan
Prior art keywords
patient
aav vector
promoter
gaa
aav
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
JP2022523856A
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English (en)
Japanese (ja)
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JP2022554141A (ja
JP2022554141A5 (https=
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/057081 external-priority patent/WO2021081338A1/en
Publication of JP2022554141A publication Critical patent/JP2022554141A/ja
Publication of JPWO2021081338A5 publication Critical patent/JPWO2021081338A5/ja
Publication of JP2022554141A5 publication Critical patent/JP2022554141A5/ja
Pending legal-status Critical Current

Links

JP2022523856A 2019-10-25 2020-10-23 糖原病を治療するための組成物及び方法 Pending JP2022554141A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962926282P 2019-10-25 2019-10-25
US62/926,282 2019-10-25
US202063083349P 2020-09-25 2020-09-25
US63/083,349 2020-09-25
PCT/US2020/057081 WO2021081338A1 (en) 2019-10-25 2020-10-23 Compositions and methods for treating glycogen storage disorders

Publications (3)

Publication Number Publication Date
JP2022554141A JP2022554141A (ja) 2022-12-28
JPWO2021081338A5 true JPWO2021081338A5 (https=) 2023-10-31
JP2022554141A5 JP2022554141A5 (https=) 2023-10-31

Family

ID=75620302

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022523856A Pending JP2022554141A (ja) 2019-10-25 2020-10-23 糖原病を治療するための組成物及び方法

Country Status (14)

Country Link
US (1) US20220387562A1 (https=)
EP (1) EP4048286A4 (https=)
JP (1) JP2022554141A (https=)
KR (1) KR20220105643A (https=)
CN (1) CN114828858A (https=)
AU (1) AU2020372429A1 (https=)
BR (1) BR112022007674A2 (https=)
CA (1) CA3158281A1 (https=)
CO (1) CO2022006772A2 (https=)
IL (1) IL292401A (https=)
MX (1) MX2022004799A (https=)
PH (1) PH12022550977A1 (https=)
TW (2) TWI885000B (https=)
WO (1) WO2021081338A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240145491A (ko) * 2022-02-03 2024-10-07 아스텔라스 진 테라피스, 인크. 폼페병의 개선된 치료를 위한 조성물 및 방법
JP2025527694A (ja) 2022-08-23 2025-08-22 マストバイオ株式会社 Ｉｌ２変異体及びそれを含むタンパク質複合体

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE437944T1 (de) * 2000-04-28 2009-08-15 Asklepios Biopharmaceutical In Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung
ATE400656T1 (de) * 2000-10-06 2008-07-15 Univ Michigan Mini-dystrophin nukleinsäure- und peptidsequenzen
RS61055B1 (sr) * 2012-06-19 2020-12-31 Univ Florida Kompozicije i postupci za lečenje bolesti
EA201890543A1 (ru) * 2015-09-17 2018-08-31 Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл Способы и материалы для генной терапии galgt2
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
WO2019012336A2 (en) * 2017-03-17 2019-01-17 Newcastle University ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY
EP3648776A4 (en) * 2017-07-06 2021-04-07 The Medical College of Wisconsin, Inc. NEW IN-VITRO AND IN-VIVO ENRICHMENT STRATEGY AGAINST LYMPHOCYTE FROM VECTOR-TRANSDUCED HSC FOR THE TREATMENT OF DISEASES
US12296026B2 (en) * 2018-02-05 2025-05-13 Astellas Gene Therapies, Inc. Transcription regulatory elements and uses thereof
EP3749771A1 (en) * 2018-02-07 2020-12-16 Genethon Hybrid regulatory elements

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