JP2016521119A - 筋ジストロフィを処置するためのエキソンスキッピング組成物 - Google Patents

筋ジストロフィを処置するためのエキソンスキッピング組成物 Download PDF

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JP2016521119A
JP2016521119A JP2016503219A JP2016503219A JP2016521119A JP 2016521119 A JP2016521119 A JP 2016521119A JP 2016503219 A JP2016503219 A JP 2016503219A JP 2016503219 A JP2016503219 A JP 2016503219A JP 2016521119 A JP2016521119 A JP 2016521119A
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antisense
antisense oligomer
acid
exon
dystrophin
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JP2016521119A5 (enExample
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リチャード ケー． ベストウィック，
リチャード ケー． ベストウィック，
ダイアン エリザベス フランク，
ダイアン エリザベス フランク，
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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
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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    • C12N15/00—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/10—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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
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    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3535—Nitrogen
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2016503219A 2013-03-14 2014-03-14 筋ジストロフィを処置するためのエキソンスキッピング組成物 Withdrawn JP2016521119A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361782706P 2013-03-14 2013-03-14
US61/782,706 2013-03-14
PCT/US2014/029766 WO2014153240A2 (en) 2013-03-14 2014-03-14 Exon skipping compositions for treating muscular dystrophy

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JP2021204872A Withdrawn JP2022031948A (ja) 2013-03-14 2021-12-17 筋ジストロフィを処置するためのエキソンスキッピング組成物
JP2024083541A Withdrawn JP2024112929A (ja) 2013-03-14 2024-05-22 筋ジストロフィを処置するためのエキソンスキッピング組成物

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JP2024083541A Withdrawn JP2024112929A (ja) 2013-03-14 2024-05-22 筋ジストロフィを処置するためのエキソンスキッピング組成物

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EP (3) EP3760720A1 (enExample)
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BR (1) BR112015023038A2 (enExample)
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Cited By (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019517605A (ja) * 2016-06-30 2019-06-24 サレプタ セラピューティクス， インコーポレイテッド ホスホロジアミダートモルホリノオリゴマーを調製するためのプロセス
JP2019518832A (ja) * 2016-05-24 2019-07-04 サレプタ セラピューティクス， インコーポレイテッド オリゴマー調製のためのプロセス
JP2019518748A (ja) * 2016-06-30 2019-07-04 サレプタ セラピューティクス， インコーポレイテッド ホスホロジアミダートモルホリノオリゴマーを調製するためのプロセス
JP2020511943A (ja) * 2016-12-19 2020-04-23 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
JP2020532504A (ja) * 2017-08-31 2020-11-12 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための方法
JP2020537501A (ja) * 2017-09-22 2020-12-24 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーのためのエクソンスキッピングオリゴマーコンジュゲート
JP2021518361A (ja) * 2018-03-16 2021-08-02 サレプタ セラピューティクス， インコーポレイテッド アンチセンス送達用キメラペプチド
JP2021521794A (ja) * 2018-04-26 2021-08-30 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およびオリゴマーコンジュゲート
JP2021526807A (ja) * 2018-06-14 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およびオリゴマーコンジュゲート
JP2023541091A (ja) * 2020-07-10 2023-09-28 チャンヂョウ シンザオール ファーマシューティカルズ カンパニー リミテッド オリゴヌクレオチドを調製するための方法
US12297219B2 (en) 2016-05-24 2025-05-13 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomers

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3808845A1 (en) 2004-06-28 2021-04-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
PT2203173E (pt) * 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
CA3066050A1 (en) 2008-10-24 2010-04-29 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for dmd
SI2499249T1 (sl) 2009-11-12 2019-02-28 The University Of Western Australia, Protismiselne molekule in postopki zdravljenja patologij
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
IL280443B (en) 2013-03-14 2022-07-01 Sarepta Therapeutics Inc Preparations that skip axon for the treatment of muscular dystrophy
MX373959B (es) 2013-03-15 2020-07-13 Sarepta Therapeutics Inc Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd).
FR3044926B1 (fr) * 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
JOP20200228A1 (ar) 2015-12-21 2017-06-16 Novartis Ag تركيبات وطرق لخفض تعبير البروتين tau
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MA45362A (fr) * 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
KR102504759B1 (ko) * 2016-05-24 2023-02-28 사렙타 쎄러퓨틱스 인코퍼레이티드 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법
CN109311919A (zh) 2016-06-30 2019-02-05 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体
CN109757108A (zh) * 2016-07-05 2019-05-14 比奥马林技术公司 具有治疗遗传疾病的改善特征的包含双环支架部分的前体mRNA剪接转换或调节寡核苷酸
US20190330626A1 (en) * 2016-07-15 2019-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for use in dystrophin transcript
CN116804031A (zh) * 2016-09-20 2023-09-26 科罗拉多州立大学董事会法人团体 使用亚磷酰胺化学法合成主链修饰的吗啉代寡核苷酸和嵌合体
MA65895B1 (fr) 2016-12-19 2024-06-28 Sarepta Therapeutics, Inc. Conjugués d'oligomères induisant le saut d'exons pour la dystrophie musculaire
MD3554553T2 (ro) 2016-12-19 2022-10-31 Sarepta Therapeutics Inc Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară
MA49986A (fr) * 2017-09-25 2020-07-01 Massachusetts Inst Technology Procédés de préparation d'oligomères morpholino phosphorodiamidate par l'intermédiaire d'une synthèse à flux rapide
EP3687519A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
US10758629B2 (en) * 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
PE20210630A1 (es) * 2018-06-26 2021-03-23 Nippon Shinyaku Co Ltd Composicion que comprende el oligonucleotido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne
CN112955153A (zh) 2018-08-02 2021-06-11 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
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
CN112912499A (zh) 2018-08-02 2021-06-04 达因疗法公司 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
CN113453723A (zh) 2018-12-07 2021-09-28 牛津大学科技创新有限公司 接头
CN113677374A (zh) * 2019-04-08 2021-11-19 国立大学法人东京医科齿科大学 肌疾病治疗用药物组合物
WO2021025899A1 (en) 2019-08-02 2021-02-11 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer pharmaceutical compositions
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
WO2022171972A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
MX2023011612A (es) 2021-03-31 2023-12-15 Entrada Therapeutics Inc Peptidos de penetracion celular ciclicos.
WO2022217366A1 (en) * 2021-04-14 2022-10-20 The Royal Institution For The Advancement Of Learning/Mcgill University Modified oligonucleotides for the treatment of duchenne muscular dystrophy
US20240358845A1 (en) 2021-05-10 2024-10-31 Entrada Therapeutics, Inc. Compositions and methods for intracellular therapeutics
IL309936A (en) 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of dystrophinopathy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4458833A1 (en) 2021-12-27 2024-11-06 Nippon Shinyaku Co., Ltd. Method for producing oligonucleic acid compound
IL315280A (en) * 2022-03-17 2024-10-01 Sarepta Therapeutics Inc Phosphorodiamidate morpholino oligomer conjugates
WO2024073588A1 (en) * 2022-09-28 2024-04-04 Board Of Regents, The University Of Texas System Respiratory dry powder delivery

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100168212A1 (en) * 2008-09-11 2010-07-01 Royal Holloway, University Of London Oligomers
WO2011057350A1 (en) * 2009-11-12 2011-05-19 The University Of Western Australia Antisense molecules and methods for treating pathologies
US8067571B2 (en) * 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
WO2012029986A1 (ja) * 2010-09-01 2012-03-08 日本新薬株式会社 アンチセンス核酸
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
WO2013112053A1 (en) * 2012-01-27 2013-08-01 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
JP2016502858A (ja) * 2012-12-20 2016-02-01 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物

Family Cites Families (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CH445129A (fr) 1964-04-29 1967-10-15 Nestle Sa Procédé pour la préparation de composés d'inclusion à poids moléculaire élevé
US3459731A (en) 1966-12-16 1969-08-05 Corn Products Co Cyclodextrin polyethers and their production
US3453257A (en) 1967-02-13 1969-07-01 Corn Products Co Cyclodextrin with cationic properties
US3426011A (en) 1967-02-13 1969-02-04 Corn Products Co Cyclodextrins with anionic properties
US3453259A (en) 1967-03-22 1969-07-01 Corn Products Co Cyclodextrin polyol ethers and their oxidation products
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
JP3022967B2 (ja) 1985-03-15 2000-03-21 アンチバイラルズ インコーポレイテッド 立体規則性ポリヌクレオチド結合ポリマー
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
KR0166088B1 (ko) 1990-01-23 1999-01-15 . 수용해도가 증가된 시클로덱스트린 유도체 및 이의 용도
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
WO1994002595A1 (en) 1992-07-17 1994-02-03 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of animal diseases
DE69435005T2 (de) 1993-05-11 2008-04-17 The University Of North Carolina At Chapel Hill Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
WO1999042091A2 (en) 1998-02-19 1999-08-26 Massachusetts Institute Of Technology Use of polycations as endosomolytic agents
US6683173B2 (en) 1998-04-03 2004-01-27 Epoch Biosciences, Inc. Tm leveling methods
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
CA2394758A1 (en) 1999-12-29 2001-07-05 A. James Mixson Histidine-containing copolymers enhance pharmaceutical agent delivery
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
DE60231975D1 (de) 2001-05-24 2009-05-28 Genzyme Corp Muskelspezifische expressionsvektoren
WO2004083432A1 (en) 2003-03-21 2004-09-30 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP3808845A1 (en) * 2004-06-28 2021-04-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
ES2852549T3 (es) * 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
CA2629323A1 (en) 2005-11-10 2007-05-24 The University Of North Carolina At Chapel Hill Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease
PL2735568T3 (pl) 2006-05-10 2018-02-28 Sarepta Therapeutics, Inc. Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami
US20070265215A1 (en) * 2006-05-11 2007-11-15 Iversen Patrick L Antisense restenosis composition and method
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
PT2203173E (pt) * 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
US8076476B2 (en) 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
KR102095478B1 (ko) 2010-05-28 2020-04-01 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
KR102339196B1 (ko) 2011-05-05 2021-12-15 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
WO2013033407A2 (en) * 2011-08-30 2013-03-07 The Regents Of The University Of California Identification of small molecules that enhance therapeutic exon skipping
CA2868174A1 (en) * 2012-03-20 2013-09-26 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8067571B2 (en) * 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US20100168212A1 (en) * 2008-09-11 2010-07-01 Royal Holloway, University Of London Oligomers
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
WO2011057350A1 (en) * 2009-11-12 2011-05-19 The University Of Western Australia Antisense molecules and methods for treating pathologies
WO2012029986A1 (ja) * 2010-09-01 2012-03-08 日本新薬株式会社 アンチセンス核酸
WO2013112053A1 (en) * 2012-01-27 2013-08-01 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
JP2016502858A (ja) * 2012-12-20 2016-02-01 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物

Non-Patent Citations (8)

* Cited by examiner, † Cited by third party
Title
FLETCHER, S. ET AL., J. GENE MED., vol. 8, JPN6018047546, 2006, pages 207 - 216, ISSN: 0003933304 *
HEEMSKERK, H.A. ET AL., J. GENE MED., vol. 11, JPN6018039246, 2009, pages 257 - 266, ISSN: 0003933300 *
MCCLOREY, G. ET AL., NEUROMUSCUL. DISORD., vol. 16, JPN6018047545, 2006, pages 583 - 590, ISSN: 0003933302 *
POPPLEWELL, L.J. ET AL., MOL. THER., vol. 17, JPN6018002658, 2009, pages 554 - 561, ISSN: 0003730257 *
POPPLEWELL, L.J. ET AL., NEUROMUSCUL. DISORD., vol. 20, JPN6018002657, 2010, pages 102 - 110, ISSN: 0003730256 *
SUMMERTON, J. AND WELLER, D., ANTISENSE NUCLEIC ACID DRUG DEV., vol. 7, no. 3, JPN6018047547, 1997, pages 187 - 195, ISSN: 0003933305 *
SWENSON, D.L. ET AL., ANTIMICROB. AGENTS CHEMOTHER., vol. 53, no. 5, JPN6018047541, 2009, pages 2089 - 2099, ISSN: 0003933301 *
中村昭則, 武田伸一, 脳と発達, vol. 42, JPN6018047544, 2010, pages 117 - 123, ISSN: 0003933303 *

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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US12297219B2 (en) 2016-05-24 2025-05-13 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomers
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