JPWO2023015304A5 - - Google Patents

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Publication number
JPWO2023015304A5
JPWO2023015304A5 JP2024501135A JP2024501135A JPWO2023015304A5 JP WO2023015304 A5 JPWO2023015304 A5 JP WO2023015304A5 JP 2024501135 A JP2024501135 A JP 2024501135A JP 2024501135 A JP2024501135 A JP 2024501135A JP WO2023015304 A5 JPWO2023015304 A5 JP WO2023015304A5
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JP
Japan
Prior art keywords
composition
intrathecal
meters
effective amount
itr
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
JP2024501135A
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English (en)
Japanese (ja)
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JP2024527742A5 (https=
JP2024527742A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/074622 external-priority patent/WO2023015304A1/en
Publication of JP2024527742A publication Critical patent/JP2024527742A/ja
Publication of JP2024527742A5 publication Critical patent/JP2024527742A5/ja
Publication of JPWO2023015304A5 publication Critical patent/JPWO2023015304A5/ja
Pending legal-status Critical Current

Links

JP2024501135A 2021-08-05 2022-08-05 アデノ随伴ウイルス粒子及びその使用方法 Pending JP2024527742A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163229936P 2021-08-05 2021-08-05
US63/229,936 2021-08-05
US202163239881P 2021-09-01 2021-09-01
US63/239,881 2021-09-01
PCT/US2022/074622 WO2023015304A1 (en) 2021-08-05 2022-08-05 Adeno-associated virus particles and methods of use thereof

Publications (3)

Publication Number Publication Date
JP2024527742A JP2024527742A (ja) 2024-07-26
JP2024527742A5 JP2024527742A5 (https=) 2025-08-14
JPWO2023015304A5 true JPWO2023015304A5 (https=) 2025-08-14

Family

ID=85156338

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024501135A Pending JP2024527742A (ja) 2021-08-05 2022-08-05 アデノ随伴ウイルス粒子及びその使用方法

Country Status (8)

Country Link
US (1) US20250041450A1 (https=)
EP (1) EP4380626A4 (https=)
JP (1) JP2024527742A (https=)
KR (1) KR20240045203A (https=)
AU (1) AU2022325165A1 (https=)
CA (1) CA3224482A1 (https=)
MX (1) MX2024001634A (https=)
WO (1) WO2023015304A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025250909A1 (en) 2024-05-31 2025-12-04 Sarepta Therapeutics, Inc. Muscle-tropic recombinant aav
CN119144658A (zh) * 2024-09-13 2024-12-17 南京鼓楼医院 一种调控平滑肌收缩的aav载体及其构建方法和应用

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9624282B2 (en) * 2012-11-26 2017-04-18 The Curators Of The University Of Missouri Microdystrophin peptides and methods for treating muscular dystrophy using the same
EP3097197B1 (en) * 2014-01-21 2020-12-16 Vrije Universiteit Brussel Muscle-specific nucleic acid regulatory elements and methods and use thereof
CA2971303C (en) * 2016-06-21 2026-03-03 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
MX2019011046A (es) * 2017-03-17 2019-10-17 Res Inst Nationwide Childrens Hospital Administracion de vectores del virus adenoasociado de microdistrofina especifica del musculo para tratar la distrofia muscular.
CA3063464A1 (en) * 2017-05-19 2018-11-22 Encoded Therapeutics, Inc. High activity regulatory elements
US20200405824A1 (en) * 2019-06-26 2020-12-31 University Of Washington Use of ribonucleotide reductase alone or in combination with micro-dystrophin to treat duchenne muscular dystrophy striated muscle disease
EP3990030A1 (en) * 2019-06-27 2022-05-04 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
AU2020368539A1 (en) * 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
WO2022029543A1 (en) * 2020-08-06 2022-02-10 Intas Pharmaceuticals Ltd. Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
CA3172481A1 (en) * 2020-11-12 2022-05-19 Precision Biosciences, Inc. Engineered meganucleases having specificity for recognition sequences in the dystrophin gene

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