JP2018530560A - デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法 - Google Patents

デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法 Download PDF

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JP2018530560A
JP2018530560A JP2018517603A JP2018517603A JP2018530560A JP 2018530560 A JP2018530560 A JP 2018530560A JP 2018517603 A JP2018517603 A JP 2018517603A JP 2018517603 A JP2018517603 A JP 2018517603A JP 2018530560 A JP2018530560 A JP 2018530560A
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exon
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myostatin
dystrophin
alkyl
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JP2018530560A5 (enExample
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ジョージ ディクソン，
ジョージ ディクソン，
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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
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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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1774—Immunoglobulin superfamily (e.g. CD2, CD4, CD8, ICAM molecules, B7 molecules, Fc-receptors, MHC-molecules)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • C07—ORGANIC CHEMISTRY
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    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
    • C07F9/65586—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system at least one of the hetero rings does not contain nitrogen as ring hetero atom
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    • 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
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    • 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
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    • 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
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    • C12N2320/00—Applications; Uses
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    • C12N2320/31—Combination therapy
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
JP2018517603A 2015-10-09 2016-10-07 デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法 Withdrawn JP2018530560A (ja)

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JP2021185544A JP2022017594A (ja) 2015-10-09 2021-11-15 デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法

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US201562239812P 2015-10-09 2015-10-09
US62/239,812 2015-10-09
PCT/US2016/056093 WO2017062835A2 (en) 2015-10-09 2016-10-07 Compositions and methods for treating duchenne muscular dystrophy and related disorders

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JP2021185544A Pending JP2022017594A (ja) 2015-10-09 2021-11-15 デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法

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BR (1) BR112018007066A2 (enExample)
EA (1) EA201890908A1 (enExample)
IL (2) IL258069A (enExample)
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US10421969B2 (en) 2011-10-04 2019-09-24 Royal Holloway And Bedford New College Oligomers
US10626396B2 (en) 2005-02-09 2020-04-21 Sarepta Therapeutics, Inc. Antisense composition and method for treating muscle atrophy
WO2020209285A1 (ja) * 2019-04-08 2020-10-15 国立大学法人東京医科歯科大学 筋疾患治療用医薬組成物
JP2020535141A (ja) * 2017-09-25 2020-12-03 サレプタ セラピューティクス， インコーポレイテッド 高速流動合成を介してホスホロジアミデートモルホリノオリゴマーを調製するための方法
US11015200B2 (en) 2015-03-18 2021-05-25 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in myostatin
JP2022511055A (ja) * 2018-12-13 2022-01-28 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コンジュゲート
JP2022516207A (ja) * 2018-12-28 2022-02-24 日本新薬株式会社 マイオスタチンシグナル阻害剤
JP2022543236A (ja) * 2019-08-02 2022-10-11 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル エクソン４４標的化核酸およびジストロフィンベースのミオパチーの治療のための当該核酸を含む組換えアデノ随伴ウイルス

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TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
AU2017290231A1 (en) * 2016-06-30 2019-02-07 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
US20180028554A1 (en) * 2016-07-05 2018-02-01 Biomarin Technologies B.V. Oligomers Having Bicyclic Scaffold Moeities
LT3554553T (lt) 2016-12-19 2022-08-25 Sarepta Therapeutics, Inc. Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos
CN117298290A (zh) * 2016-12-19 2023-12-29 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体缀合物
EP3554552B1 (en) * 2016-12-19 2022-08-17 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
US20200131231A1 (en) 2017-02-17 2020-04-30 Oxford University Innovation Limited Cell penetrating peptides
SG11202001074RA (en) * 2017-08-31 2020-03-30 Sarepta Therapeutics Inc Methods for treating muscular dystrophy
EA201991450A1 (ru) * 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
WO2019108065A1 (en) 2017-12-01 2019-06-06 Merus N.V. Use of bispecific antibody and il-15 for combination therapy
KR20200116933A (ko) * 2018-01-31 2020-10-13 더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템 인간 심근세포에서 디스트로핀 돌연변이를 교정하기 위한 조성물 및 방법
IL277889B2 (en) 2018-04-12 2025-01-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods of using them
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US10758629B2 (en) * 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
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JP2021526796A (ja) * 2018-06-13 2021-10-11 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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JP7611825B2 (ja) 2018-08-02 2025-01-10 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それらの使用
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating 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
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CN113383078A (zh) * 2018-12-06 2021-09-10 波涛生命科学有限公司 寡核苷酸组合物及其方法
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WO2023212193A1 (en) * 2022-04-27 2023-11-02 The Curators Of The University Of Missouri Micro-dystrophin for heart protection
IL327379A (en) * 2023-09-26 2026-05-01 Entrada Therapeutics Inc Compounds and methods for skipping exon 50 in Duchenne muscular dystrophy
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