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
Application number
JP2020557910A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021521794A (ja
JPWO2019209764A5 (https=
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
Publication of JPWO2019209764A5 publication Critical patent/JPWO2019209764A5/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 (3)

Publication Number Publication Date
JP2021521794A JP2021521794A (ja) 2021-08-30
JP2021521794A5 true JP2021521794A5 (https=) 2022-04-25
JPWO2019209764A5 JPWO2019209764A5 (https=) 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 (https=)
EP (1) EP3784248A4 (https=)
JP (2) JP2021521794A (https=)
WO (1) WO2019209764A2 (https=)

Families Citing this family (7)

* 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
CA3211038A1 (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
ATE498685T1 (de) * 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
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
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
WO2012150960A1 (en) * 2011-05-05 2012-11-08 Avi Biopharma, 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
CA2894899A1 (en) * 2012-12-20 2014-06-26 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
RS58529B1 (sr) * 2013-03-14 2019-04-30 Sarepta Therapeutics Inc Sastavi koji preskaču egzon za tretiranje mišićne distrofije
CA2906812A1 (en) * 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
WO2016138534A2 (en) * 2015-02-27 2016-09-01 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
MA49775A (fr) * 2016-05-24 2020-06-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
TW201811807A (zh) * 2016-06-30 2018-04-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物

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