JP2020537497A - エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法 - Google Patents

エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法 Download PDF

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JP2020537497A
JP2020537497A JP2020514269A JP2020514269A JP2020537497A JP 2020537497 A JP2020537497 A JP 2020537497A JP 2020514269 A JP2020514269 A JP 2020514269A JP 2020514269 A JP2020514269 A JP 2020514269A JP 2020537497 A JP2020537497 A JP 2020537497A
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polynucleic acid
acid molecule
examples
exon
conjugate according
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JP2020537497A5 (enExample
Inventor
アーサー エー レビン，
アーサー エー レビン，
ジョン ジアル，アンドリュー
ジョン ジアル，アンドリュー
カラミアン コクラン，マイケル
カラミアン コクラン，マイケル
ホゥアン，ハンホア
ラマナ ドッパラプディ，ベンカタ
ラマナ ドッパラプディ，ベンカタ
バーク，ロブ
ダイアナ ダリモント，ベアトリス
ダイアナ ダリモント，ベアトリス
シ，ユンユ
ジョンズ，レイチェル
Original Assignee
アビディティー バイオサイエンシーズ，インク．
アビディティー バイオサイエンシーズ，インク．
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Publication of JP2020537497A publication Critical patent/JP2020537497A/ja
Publication of JP2020537497A5 publication Critical patent/JP2020537497A5/ja
Priority to JP2023118556A priority Critical patent/JP7635307B2/ja
Priority to JP2025021008A priority patent/JP2025069453A/ja
Pending legal-status Critical Current

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    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
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    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6843—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a material from animals or humans
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6949—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit inclusion complexes, e.g. clathrates, cavitates or fullerenes
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/3233—Morpholino-type ring
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    • C12N2320/00—Applications; Uses
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    • C12N2320/33—Alteration of splicing

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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
JP2020514269A 2017-09-22 2018-09-21 エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法 Pending JP2020537497A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023118556A JP7635307B2 (ja) 2017-09-22 2023-07-20 エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法
JP2025021008A JP2025069453A (ja) 2017-09-22 2025-02-12 エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762561939P 2017-09-22 2017-09-22
US62/561,939 2017-09-22
US201862696766P 2018-07-11 2018-07-11
US62/696,766 2018-07-11
PCT/US2018/052289 WO2019060775A1 (en) 2017-09-22 2018-09-21 NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND EXON JUMP INDUCTION METHODS

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JP2020537497A5 JP2020537497A5 (enExample) 2021-10-21

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JP2023118556A Active JP7635307B2 (ja) 2017-09-22 2023-07-20 エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法
JP2025021008A Pending JP2025069453A (ja) 2017-09-22 2025-02-12 エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法

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JP2025021008A Pending JP2025069453A (ja) 2017-09-22 2025-02-12 エクソンスキッピングを誘発する核酸ポリペプチド組成物と方法

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US (2) US20200282074A1 (enExample)
EP (1) EP3684376A4 (enExample)
JP (3) JP2020537497A (enExample)
KR (2) KR20250065737A (enExample)
CN (1) CN111770757A (enExample)
AU (2) AU2018335880B2 (enExample)
CA (1) CA3075425A1 (enExample)
IL (1) IL273429A (enExample)
MA (1) MA50269A (enExample)
MX (1) MX2020003130A (enExample)
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WO (1) WO2019060775A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023026994A1 (ja) * 2021-08-21 2023-03-02 武田薬品工業株式会社 ヒトトランスフェリンレセプター結合ペプチド－薬物コンジュゲート
JP2025511714A (ja) * 2022-04-05 2025-04-16 アビディティー バイオサイエンシーズ，インク． 抗体オリゴヌクレオチドコンジュゲート組成物、およびｄｍｄエクソン４４スキッピングを誘導する方法

Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
WO2018129384A1 (en) 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
CN118667812A (zh) 2017-12-06 2024-09-20 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
JP2021533200A (ja) 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US20210308273A1 (en) 2018-08-02 2021-10-07 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
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
KR20220038771A (ko) * 2019-08-02 2022-03-29 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 엑손 44-표적화된 핵산 및 상기 핵산을 포함하는 디스트로핀-기반 근병증 치료용 재조합 아데노-관련 바이러스
CN114929336A (zh) * 2019-09-19 2022-08-19 阿尔奈科学有限责任公司 用于调节基因剪接的化合物和方法
EP4079329A4 (en) * 2019-12-19 2024-04-17 Nippon Shinyaku Co., Ltd. Antisense nucleic acid enabling exon skipping
US20230088865A1 (en) * 2020-01-10 2023-03-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4121063A4 (en) 2020-03-19 2024-07-03 Avidity Biosciences, Inc. COMPOSITIONS AND METHODS FOR TREATING FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
JP7748962B2 (ja) 2020-03-27 2025-10-03 アビディティー バイオサイエンシーズ，インク． 筋ジストロフィーを処置するための組成物および方法
JP2023535073A (ja) * 2020-07-23 2023-08-15 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
KR20230117182A (ko) * 2020-12-04 2023-08-07 다인 세라퓨틱스, 인크. 항체-올리고뉴클레오티드 복합체 및 그의 용도
AU2022267324A1 (en) 2021-04-30 2023-12-14 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
AR125992A1 (es) * 2021-05-28 2023-08-30 Novo Nordisk As Composiciones y métodos para inhibir la expresión del componente 1 de reducción de amidoxima mitocondrial (marc1)
JP2024525613A (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 筋標的化複合体およびジストロフィノパチーを処置するためのその使用
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11648318B2 (en) 2021-07-09 2023-05-16 Dyne Therapeutics, Inc. Anti-transferrin receptor (TFR) antibody and uses thereof
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
EP4366784A1 (en) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
CN118355035A (zh) * 2021-07-09 2024-07-16 达因疗法公司 肌肉靶向复合物及其用于治疗肌养蛋白病的用途
KR20240055874A (ko) 2021-09-16 2024-04-29 어비디티 바이오사이언시스 인크. 안면견갑상완 근이영양증을 치료하는 조성물 및 방법
WO2023122347A2 (en) * 2021-12-23 2023-06-29 Mirecule, Inc. Compositions for delivery of polynucleotides
EP4466360A1 (en) * 2022-01-20 2024-11-27 Bolden Therapeutics, Inc. Musk-targeting oligonucleotides
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
EP4493693A1 (en) 2022-03-17 2025-01-22 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
KR20250004770A (ko) 2022-04-15 2025-01-08 다인 세라퓨틱스, 인크. 근긴장성 이영양증을 치료하기 위한 근육 표적화 복합체 및 제제
EP4617301A1 (en) 2022-11-07 2025-09-17 Nippon Shokubai Co., Ltd. Method for producing solubilizing polymer and method for producing water absorbent resin
WO2024197302A1 (en) * 2023-03-23 2024-09-26 Yale University Compositions and methods for delivering antibody oligonucleotide conjugates for exon skipping
CN116381125A (zh) * 2023-06-05 2023-07-04 迦进生物医药(上海)有限公司 评估与蛋白偶联的核酸稳定性的方法及试剂盒
WO2025072246A1 (en) * 2023-09-26 2025-04-03 Entrada Therapeutics, Inc. Compounds and methods for skipping exon 50 in duchenne muscular dystrophy
US20250325694A1 (en) * 2024-04-03 2025-10-23 Avidity Biosciences, Inc. Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 52 skipping

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT2203173E (pt) * 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
AU2009234266B2 (en) * 2008-04-11 2015-08-06 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
EP2119783A1 (en) * 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US20110130346A1 (en) * 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
NZ732507A (en) * 2013-03-15 2018-08-31 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
EP3159409B1 (en) * 2014-06-17 2019-12-04 Nippon Shinyaku Co., Ltd. Antisense nucleic acid for use in the treatment of duchenne's muscular dystrophy
NZ737757A (en) * 2015-05-19 2023-07-28 Sarepta Therapeutics Inc Peptide oligonucleotide conjugates
WO2018129384A1 (en) * 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
US12071621B2 (en) * 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
JOURNAL OF CONTROLLED RELEASE, 2016, VOL.237, PP.1-13, JPN6022031293, ISSN: 0005120988 *
PHARMACEUTICAL RESEARCH,1995, VOL.12, NO.10, PP.1548-1553, JPN6022031290, ISSN: 0005120989 *
脳と発達, 2010, VOL.42,PP.117-123, JPN6022031287, ISSN: 0005120990 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023026994A1 (ja) * 2021-08-21 2023-03-02 武田薬品工業株式会社 ヒトトランスフェリンレセプター結合ペプチド－薬物コンジュゲート
JP2025511714A (ja) * 2022-04-05 2025-04-16 アビディティー バイオサイエンシーズ，インク． 抗体オリゴヌクレオチドコンジュゲート組成物、およびｄｍｄエクソン４４スキッピングを誘導する方法

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