JPWO2019241385A5 - - Google Patents

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Publication number
JPWO2019241385A5
JPWO2019241385A5 JP2020567764A JP2020567764A JPWO2019241385A5 JP WO2019241385 A5 JPWO2019241385 A5 JP WO2019241385A5 JP 2020567764 A JP2020567764 A JP 2020567764A JP 2020567764 A JP2020567764 A JP 2020567764A JP WO2019241385 A5 JPWO2019241385 A5 JP WO2019241385A5
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JP
Japan
Prior art keywords
seq
antisense oligomer
pharmaceutically acceptable
acceptable salt
formula
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
JP2020567764A
Other languages
English (en)
Japanese (ja)
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JP2021526796A (ja
JP2021526796A5 (https=
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/036764 external-priority patent/WO2019241385A2/en
Publication of JP2021526796A publication Critical patent/JP2021526796A/ja
Publication of JP2021526796A5 publication Critical patent/JP2021526796A5/ja
Publication of JPWO2019241385A5 publication Critical patent/JPWO2019241385A5/ja
Priority to JP2024096569A priority Critical patent/JP2024116362A/ja
Withdrawn legal-status Critical Current

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JP2020567764A 2018-06-13 2019-06-12 筋ジストロフィーに対するエクソンスキッピングオリゴマー Withdrawn JP2021526796A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024096569A JP2024116362A (ja) 2018-06-13 2024-06-14 筋ジストロフィーに対するエクソンスキッピングオリゴマー

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862684615P 2018-06-13 2018-06-13
US62/684,615 2018-06-13
US201962860446P 2019-06-12 2019-06-12
PCT/US2019/036764 WO2019241385A2 (en) 2018-06-13 2019-06-12 Exon skipping oligomers for muscular dystropy
US62/860,446 2019-06-12

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024096569A Division JP2024116362A (ja) 2018-06-13 2024-06-14 筋ジストロフィーに対するエクソンスキッピングオリゴマー

Publications (3)

Publication Number Publication Date
JP2021526796A JP2021526796A (ja) 2021-10-11
JP2021526796A5 JP2021526796A5 (https=) 2022-06-13
JPWO2019241385A5 true JPWO2019241385A5 (https=) 2022-06-13

Family

ID=68843196

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020567764A Withdrawn JP2021526796A (ja) 2018-06-13 2019-06-12 筋ジストロフィーに対するエクソンスキッピングオリゴマー
JP2024096569A Pending JP2024116362A (ja) 2018-06-13 2024-06-14 筋ジストロフィーに対するエクソンスキッピングオリゴマー

Family Applications After (1)

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JP2024096569A Pending JP2024116362A (ja) 2018-06-13 2024-06-14 筋ジストロフィーに対するエクソンスキッピングオリゴマー

Country Status (3)

Country Link
US (2) US20220251551A1 (https=)
JP (2) JP2021526796A (https=)
WO (1) WO2019241385A2 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP3829595A4 (en) 2018-08-02 2022-08-24 Dyne Therapeutics, Inc. MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES
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
WO2021150867A1 (en) * 2020-01-24 2021-07-29 Sarepta Therapeutics, Inc. Designing antisense oligonucleotide delivery peptides by interpretable machine learning
US20230140736A1 (en) 2020-02-28 2023-05-04 Nippon Shinyaku Co., Ltd. Antisense nucleic acid inducing skipping of exon 51
EP3978608A1 (en) * 2020-10-05 2022-04-06 SQY Therapeutics Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
IL309936A (en) 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of dystrophinopathy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
AR128918A1 (es) * 2022-03-30 2024-06-26 Biomarin Pharm Inc Oligonucleótidos que omiten el exón 51 de distrofina y sus aplicaciones

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE498685T1 (de) * 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
AU2008317566B2 (en) * 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
WO2009101399A1 (en) * 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)
CN112574988A (zh) * 2008-10-24 2021-03-30 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
JP5786109B2 (ja) * 2008-10-27 2015-09-30 プロセンサ テクノロジーズ ベー．フェー． Ｄｕｃｈｅｎｎｅ型筋ジストロフィーｍＲＮＡ前駆体のエクソン４５の効率的なスキッピングのための方法及び手段
CA2759899A1 (en) * 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
LT2499249T (lt) * 2009-11-12 2018-12-27 The University Of Western Australia Priešprasmės molekulės ir patologijų gydymo būdai
EP2672977A1 (en) * 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
WO2012150960A1 (en) * 2011-05-05 2012-11-08 Avi Biopharma, Inc. Peptide oligonucleotide conjugates
CN112251436A (zh) * 2012-01-27 2021-01-22 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
NZ731587A (en) * 2013-03-14 2021-07-30 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
CA2906812A1 (en) * 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
EP3858993A1 (en) * 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
US20190275072A1 (en) * 2016-05-24 2019-09-12 Sarepta Therapeutics, Inc. Pharmaceutical compositions comprising eteplirsen
US20190330626A1 (en) * 2016-07-15 2019-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for use in dystrophin transcript
GB201711809D0 (en) * 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide

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