JP2021526796A - 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 - Google Patents

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 Download PDF

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JP2021526796A
JP2021526796A JP2020567764A JP2020567764A JP2021526796A JP 2021526796 A JP2021526796 A JP 2021526796A JP 2020567764 A JP2020567764 A JP 2020567764A JP 2020567764 A JP2020567764 A JP 2020567764A JP 2021526796 A JP2021526796 A JP 2021526796A
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seq
pharmaceutically acceptable
antisense oligomer
acceptable salt
formula
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JP2021526796A5 (https=
JPWO2019241385A5 (https=
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フレデリック ジョゼフ シュネル，
マルコ パッシーニ，
ネイサ エストレラ，
ガンナー ハンソン，
ミン ジョウ，
リチャード ベストウィック，
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サレプタ セラピューティクス， インコーポレイテッド
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Publication of JP2021526796A5 publication Critical patent/JP2021526796A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
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)

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

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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

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

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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

Citations (10)

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WO2006000057A1 (en) * 2004-06-28 2006-01-05 SMITHKLINE BEECHAM CORPORATION, doing business as GLAXOSMITHKLINE Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2009101399A1 (en) * 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)
JP2012506703A (ja) * 2008-10-24 2012-03-22 エイブイアイ バイオファーマ， インコーポレイテッド Ｄｍｄのための複数のエキソンスキッピング組成物
JP2012506698A (ja) * 2008-10-27 2012-03-22 アカデミシュ ジーケンハウス ライデン ヒトＤｕｃｈｅｎｎｅ型筋ジストロフィー遺伝子のエクソン４３、４６、５０〜５３のうちの少なくとも１個の効率的なスキッピングのための方法及び手段
JP2012524540A (ja) * 2009-04-24 2012-10-18 プロセンサ テクノロジーズ ビー．ブイ． Ｄｍｄを処置するためのイノシンを含むオリゴヌクレオチド
JP2014507143A (ja) * 2011-02-08 2014-03-27 ザ シャーロット−メクレンバーグ ホスピタル オーソリティ ドゥーイング／ビジネス／アズ キャロライナズ ヘルスケア システム アンチセンスオリゴヌクレオチド
JP2014515762A (ja) * 2011-05-05 2014-07-03 サレプタ セラピューティクス， インコーポレイテッド ペプチドオリゴヌクレオチドコンジュゲート
JP2015509922A (ja) * 2012-01-27 2015-04-02 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド
WO2017062835A2 (en) * 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
WO2018014042A1 (en) * 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dystrophin transcript

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AU2008317566B2 (en) * 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
LT2499249T (lt) * 2009-11-12 2018-12-27 The University Of Western Australia Priešprasmės molekulės ir patologijų gydymo būdai
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
US20190275072A1 (en) * 2016-05-24 2019-09-12 Sarepta Therapeutics, Inc. Pharmaceutical compositions comprising eteplirsen
GB201711809D0 (en) * 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide

Patent Citations (10)

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WO2006000057A1 (en) * 2004-06-28 2006-01-05 SMITHKLINE BEECHAM CORPORATION, doing business as GLAXOSMITHKLINE Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2009101399A1 (en) * 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)
JP2012506703A (ja) * 2008-10-24 2012-03-22 エイブイアイ バイオファーマ， インコーポレイテッド Ｄｍｄのための複数のエキソンスキッピング組成物
JP2012506698A (ja) * 2008-10-27 2012-03-22 アカデミシュ ジーケンハウス ライデン ヒトＤｕｃｈｅｎｎｅ型筋ジストロフィー遺伝子のエクソン４３、４６、５０〜５３のうちの少なくとも１個の効率的なスキッピングのための方法及び手段
JP2012524540A (ja) * 2009-04-24 2012-10-18 プロセンサ テクノロジーズ ビー．ブイ． Ｄｍｄを処置するためのイノシンを含むオリゴヌクレオチド
JP2014507143A (ja) * 2011-02-08 2014-03-27 ザ シャーロット−メクレンバーグ ホスピタル オーソリティ ドゥーイング／ビジネス／アズ キャロライナズ ヘルスケア システム アンチセンスオリゴヌクレオチド
JP2014515762A (ja) * 2011-05-05 2014-07-03 サレプタ セラピューティクス， インコーポレイテッド ペプチドオリゴヌクレオチドコンジュゲート
JP2015509922A (ja) * 2012-01-27 2015-04-02 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド
WO2017062835A2 (en) * 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
WO2018014042A1 (en) * 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dystrophin transcript

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Title
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US20250290069A1 (en) 2025-09-18
WO2019241385A3 (en) 2020-01-23
WO2019241385A2 (en) 2019-12-19
US20220251551A1 (en) 2022-08-11
JP2024116362A (ja) 2024-08-27

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