JPWO2022119979A5 - - Google Patents

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Publication number
JPWO2022119979A5
JPWO2022119979A5 JP2023533279A JP2023533279A JPWO2022119979A5 JP WO2022119979 A5 JPWO2022119979 A5 JP WO2022119979A5 JP 2023533279 A JP2023533279 A JP 2023533279A JP 2023533279 A JP2023533279 A JP 2023533279A JP WO2022119979 A5 JPWO2022119979 A5 JP WO2022119979A5
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JP
Japan
Prior art keywords
aav
vector
capsid protein
seq
protein
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
JP2023533279A
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English (en)
Japanese (ja)
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JP2023551874A5 (https=
JP2023551874A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/061490 external-priority patent/WO2022119979A1/en
Publication of JP2023551874A publication Critical patent/JP2023551874A/ja
Publication of JP2023551874A5 publication Critical patent/JP2023551874A5/ja
Publication of JPWO2022119979A5 publication Critical patent/JPWO2022119979A5/ja
Pending legal-status Critical Current

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JP2023533279A 2020-12-01 2021-12-01 筋強直性ジストロフィー1型を処置するためのrna標的化組成物および方法 Pending JP2023551874A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063119977P 2020-12-01 2020-12-01
US63/119,977 2020-12-01
US202063130092P 2020-12-23 2020-12-23
US63/130,092 2020-12-23
US202163278746P 2021-11-12 2021-11-12
US63/278,746 2021-11-12
PCT/US2021/061490 WO2022119979A1 (en) 2020-12-01 2021-12-01 Rna-targeting compositions and methods for treating myotonic dystrophy type 1

Publications (3)

Publication Number Publication Date
JP2023551874A JP2023551874A (ja) 2023-12-13
JP2023551874A5 JP2023551874A5 (https=) 2024-12-09
JPWO2022119979A5 true JPWO2022119979A5 (https=) 2024-12-09

Family

ID=79731176

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023533279A Pending JP2023551874A (ja) 2020-12-01 2021-12-01 筋強直性ジストロフィー1型を処置するためのrna標的化組成物および方法

Country Status (7)

Country Link
US (1) US20240108751A1 (https=)
EP (1) EP4255471A1 (https=)
JP (1) JP2023551874A (https=)
KR (1) KR20230129162A9 (https=)
AU (1) AU2021391645A1 (https=)
CA (1) CA3200588A1 (https=)
WO (1) WO2022119979A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
IL301675A (en) 2020-10-09 2023-05-01 Tenaya Therapeutics Inc Methods and preparations for placophilin-2 gene therapy
KR20230127221A9 (ko) * 2020-12-01 2024-12-06 로카나바이오 인크. Cag 반복 질환을 치료하기 위한 rna 표적화 조성물 및 방법
WO2022221278A1 (en) * 2021-04-12 2022-10-20 Locanabio, Inc. Compositions and methods comprising hybrid promoters
GB202207026D0 (en) * 2022-05-13 2022-06-29 Univ Oxford Innovation Ltd Method
WO2025101968A1 (en) * 2023-11-10 2025-05-15 Repeat Therapeutics, Inc. Treatment of neurodegenerative diseases caused by repeat expansion with nucleoside administration
WO2025155722A1 (en) 2024-01-16 2025-07-24 Astellas Gene Therapies, Inc. Muscle selective hybrid regulatory combinations and methods of use thereof for the treatment of myotonic dystrophy type 1
WO2026015609A1 (en) * 2024-07-10 2026-01-15 The Johns Hopkins University Vectors and methods for increased non-coding rna expression

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
DK2191001T3 (en) 2007-04-09 2016-09-19 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
CN101895633A (zh) 2010-07-14 2010-11-24 中兴通讯股份有限公司 一种移动终端及其解锁方法
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
WO2013058404A1 (ja) 2011-10-21 2013-04-25 国立大学法人九州大学 Pprモチーフを利用したrna結合性蛋白質の設計方法及びその利用
US10330674B2 (en) 2015-01-13 2019-06-25 Massachusetts Institute Of Technology Pumilio domain-based modular protein architecture for RNA binding
US10476825B2 (en) 2017-08-22 2019-11-12 Salk Institue for Biological Studies RNA targeting methods and compositions
JP2021526858A (ja) * 2018-06-08 2021-10-11 ロックアネイビオ, インコーポレイテッド Rna標的化融合タンパク質組成物および使用方法
KR20230127221A9 (ko) * 2020-12-01 2024-12-06 로카나바이오 인크. Cag 반복 질환을 치료하기 위한 rna 표적화 조성물 및 방법

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