JP2021521794A5 - - Google Patents

Info

Publication number
JP2021521794A5
JP2021521794A5 JP2020557910A JP2020557910A JP2021521794A5 JP 2021521794 A5 JP2021521794 A5 JP 2021521794A5 JP 2020557910 A JP2020557910 A JP 2020557910A JP 2020557910 A JP2020557910 A JP 2020557910A JP 2021521794 A5 JP2021521794 A5 JP 2021521794A5
Authority
JP
Japan
Prior art keywords
pharmaceutically acceptable
antisense oligomer
acceptable salt
exon
dystrophin
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
JP2020557910A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021521794A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/028613 external-priority patent/WO2019209764A2/en
Publication of JP2021521794A publication Critical patent/JP2021521794A/ja
Publication of JP2021521794A5 publication Critical patent/JP2021521794A5/ja
Priority to JP2023202664A priority Critical patent/JP2024009344A/ja
Pending legal-status Critical Current

Links

JP2020557910A 2018-04-26 2019-04-23 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およびオリゴマーコンジュゲート Pending JP2021521794A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023202664A JP2024009344A (ja) 2018-04-26 2023-11-30 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およびオリゴマーコンジュゲート

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862663162P 2018-04-26 2018-04-26
US62/663,162 2018-04-26
PCT/US2019/028613 WO2019209764A2 (en) 2018-04-26 2019-04-23 Exon skipping oligomers and oligomer conjugates for muscular dystrophy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023202664A Division JP2024009344A (ja) 2018-04-26 2023-11-30 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およびオリゴマーコンジュゲート

Publications (2)

Publication Number Publication Date
JP2021521794A JP2021521794A (ja) 2021-08-30
JP2021521794A5 true JP2021521794A5 (enExample) 2022-04-25

Family

ID=68295765

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020557910A Pending JP2021521794A (ja) 2018-04-26 2019-04-23 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およびオリゴマーコンジュゲート
JP2023202664A Pending JP2024009344A (ja) 2018-04-26 2023-11-30 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およびオリゴマーコンジュゲート

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023202664A Pending JP2024009344A (ja) 2018-04-26 2023-11-30 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およびオリゴマーコンジュゲート

Country Status (4)

Country Link
US (2) US20210102205A1 (enExample)
EP (1) EP3784248A4 (enExample)
JP (2) JP2021521794A (enExample)
WO (1) WO2019209764A2 (enExample)

Families Citing this family (7)

* 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
WO2022171972A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
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

Family Cites Families (12)

* 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
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
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
CA3092114A1 (en) * 2011-05-05 2012-11-08 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
CN103998458B (zh) * 2011-08-30 2018-10-09 医学研究理事会 具有中央疏水域的细胞穿透肽
HK1216902A1 (zh) * 2012-12-20 2016-12-09 Sarepta Therapeutics, Inc. 用作治疗肌肉萎缩的改进外显子跳跃组合物
US20140315977A1 (en) * 2013-03-14 2014-10-23 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
US20140329762A1 (en) * 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
EP3262056A4 (en) * 2015-02-27 2018-09-19 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
EP3858993A1 (en) * 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
BR112018074346B1 (pt) * 2016-05-24 2023-01-03 Sarepta Therapeutics, Inc. Composto oligomérico e processo para preparar um composto oligomérico
JP2019525742A (ja) * 2016-06-30 2019-09-12 サレプタ セラピューティクス, インコーポレイテッド 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

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