JP2021526796A5 - - Google Patents

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Publication number
JP2021526796A5
JP2021526796A5 JP2020567764A JP2020567764A JP2021526796A5 JP 2021526796 A5 JP2021526796 A5 JP 2021526796A5 JP 2020567764 A JP2020567764 A JP 2020567764A JP 2020567764 A JP2020567764 A JP 2020567764A JP 2021526796 A5 JP2021526796 A5 JP 2021526796A5
Authority
JP
Japan
Prior art keywords
seq
pharmaceutically acceptable
antisense oligomer
acceptable salt
oligomer according
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.)
Withdrawn
Application number
JP2020567764A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021526796A (ja
Filing date
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
Priority to JP2024096569A priority Critical patent/JP2024116362A/ja
Withdrawn legal-status Critical Current

Links

JP2020567764A 2018-06-13 2019-06-12 筋ジストロフィーに対するエクソンスキッピングオリゴマー Withdrawn JP2021526796A (ja)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 (2)

Publication Number Publication Date
JP2021526796A JP2021526796A (ja) 2021-10-11
JP2021526796A5 true JP2021526796A5 (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JP2020567764A Withdrawn JP2021526796A (ja) 2018-06-13 2019-06-12 筋ジストロフィーに対するエクソンスキッピングオリゴマー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)

Application Number Title Priority Date Filing Date
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
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
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
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CA3108282A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2021150867A1 (en) * 2020-01-24 2021-07-29 Sarepta Therapeutics, Inc. Designing antisense oligonucleotide delivery peptides by interpretable machine learning
IL295967A (en) 2020-02-28 2022-10-01 Nippon Shinyaku Co Ltd Antisense nucleic acids for splicing on 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
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス，インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11638761B2 (en) 2021-07-09 2023-05-02 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
EP2933332A1 (en) * 2004-06-28 2015-10-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
ES2639852T3 (es) * 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
WO2009101399A1 (en) * 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)
BR122020021379B1 (pt) * 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
AU2009310557B2 (en) * 2008-10-27 2014-09-11 Academisch Ziekenhuis Leiden Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA
EP2421971B1 (en) * 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
TR201816523T4 (tr) * 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
EP2672977A1 (en) * 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
CA3092114A1 (en) * 2011-05-05 2012-11-08 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
NZ627896A (en) * 2012-01-27 2016-11-25 Biomarin Technologies B V Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
CN110218727A (zh) * 2013-03-14 2019-09-10 萨勒普塔医疗公司 用于治疗肌营养不良的外显子跳跃组合物
US20140329762A1 (en) * 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. 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
AU2017278699A1 (en) * 2016-05-24 2018-11-15 Sarepta Therapeutics, Inc. Pharmaceutical composition 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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