JP2023551279A5 - - Google Patents

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Publication number
JP2023551279A5
JP2023551279A5 JP2023532456A JP2023532456A JP2023551279A5 JP 2023551279 A5 JP2023551279 A5 JP 2023551279A5 JP 2023532456 A JP2023532456 A JP 2023532456A JP 2023532456 A JP2023532456 A JP 2023532456A JP 2023551279 A5 JP2023551279 A5 JP 2023551279A5
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JP
Japan
Application number
JP2023532456A
Other languages
Japanese (ja)
Other versions
JPWO2022115745A5 (https=
JP2023551279A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/061109 external-priority patent/WO2022115745A1/en
Publication of JP2023551279A publication Critical patent/JP2023551279A/ja
Publication of JPWO2022115745A5 publication Critical patent/JPWO2022115745A5/ja
Publication of JP2023551279A5 publication Critical patent/JP2023551279A5/ja
Pending legal-status Critical Current

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JP2023532456A 2020-11-30 2021-11-30 顔面肩甲上腕筋ジストロフィー(fshd)を治療するための組成物及び方法 Pending JP2023551279A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063119268P 2020-11-30 2020-11-30
US63/119,268 2020-11-30
PCT/US2021/061109 WO2022115745A1 (en) 2020-11-30 2021-11-30 Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)

Publications (3)

Publication Number Publication Date
JP2023551279A JP2023551279A (ja) 2023-12-07
JPWO2022115745A5 JPWO2022115745A5 (https=) 2024-11-14
JP2023551279A5 true JP2023551279A5 (https=) 2024-11-14

Family

ID=79021122

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023532456A Pending JP2023551279A (ja) 2020-11-30 2021-11-30 顔面肩甲上腕筋ジストロフィー(fshd)を治療するための組成物及び方法

Country Status (8)

Country Link
US (1) US20240026356A1 (https=)
EP (1) EP4251752A1 (https=)
JP (1) JP2023551279A (https=)
KR (1) KR20230128470A (https=)
AU (1) AU2021385595A1 (https=)
CA (1) CA3203585A1 (https=)
IL (1) IL303230A (https=)
WO (1) WO2022115745A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
AU2019419494B2 (en) * 2018-12-31 2026-03-26 Research Institute At Nationwide Children's Hospital DUX4 RNA silencing using RNA targeting CRISPR-Cas13b
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JP2025507958A (ja) 2022-03-04 2025-03-21 ロックアネイビオ, インコーポレイテッド 操作された核内低分子RNA(snRNA)を含む組成物および方法
WO2025225719A1 (en) * 2024-04-26 2025-10-30 Chugai Seiyaku Kabushiki Kaisha Treatment and prevention of muscular dystrophy and its related diseases or disorders with satralizumab

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PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
AU707866B2 (en) 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
AU722196B2 (en) 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
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EP0932418B1 (en) 1996-09-06 2007-12-05 The Trustees Of The University Of Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
JP2001514845A (ja) 1997-09-05 2001-09-18 ターゲティッド ジェネティクス コーポレイション 組換えaavベクターの高力価ヘルパーなし調製物を生成するための方法
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
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AU2012286994B2 (en) 2011-07-25 2017-08-10 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of DUX4
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
JP6202701B2 (ja) 2014-03-21 2017-09-27 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及びプログラム
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
AU2017222653B2 (en) * 2016-02-26 2023-06-01 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inducing DUX4 exon skipping
WO2020028134A1 (en) * 2018-07-30 2020-02-06 Fred Hutchinson Cancer Research Center Methods and compositions for treating cancer
AU2019419494B2 (en) * 2018-12-31 2026-03-26 Research Institute At Nationwide Children's Hospital DUX4 RNA silencing using RNA targeting CRISPR-Cas13b

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