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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    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C12N2310/353—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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Physics & Mathematics (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • 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)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (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) EP3495485A3 (enExample)
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KR (5) KR20240094032A (enExample)
CN (2) CN105392884B (enExample)
AU (3) AU2014236160A1 (enExample)
BR (1) BR112015023038A2 (enExample)
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SI (1) SI2970964T1 (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
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
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
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
DK2206781T3 (en) 2004-06-28 2016-03-07 Univ Western Australia The antisense oligonucleotides in inducing exon-skipping and methods of use thereof
ES2914775T3 (es) * 2007-10-26 2022-06-16 Academisch Ziekenhuis Leiden Medios y métodos para contrarresta trastornos del músculo
IL304042A (en) 2008-10-24 2023-08-01 Sarepta Therapeutics Inc Antisense compounds for inducing axon termination and preparations containing them for the treatment of muscular dystrophy
IL297299A (en) 2009-11-12 2022-12-01 Univ Western Australia Antisense oligonucleotides for inducing exon skipping in the dystrophin gene
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
KR20250047401A (ko) 2013-03-14 2025-04-03 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
CA2906812A1 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
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
CA3024182A1 (en) * 2016-05-24 2017-11-30 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
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
WO2018005805A1 (en) * 2016-06-30 2018-01-04 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
EP4252845A3 (en) * 2016-07-05 2024-05-22 BioMarin Technologies B.V. Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders
EP3485015A4 (en) * 2016-07-15 2020-07-29 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS OF MODULATION OF DYSTROPHINE TRANSCRIPT
EP3515445A4 (en) 2016-09-20 2020-05-27 The Regents of The University of Colorado, A Body Corporate SYNTHESIS OF BACKBONE-MODIFIED MORPHOLINO-OLIGONUCLEOTIDES AND CHIMERES WITH THE AID OF PHOSPHORAMIDITE CHEMISTRY
PT3554553T (pt) 2016-12-19 2022-08-04 Sarepta Therapeutics Inc Conjugados oligoméricos de salto de exão para a distrofia muscular
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
JP7218361B2 (ja) * 2017-09-25 2023-02-06 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
US20200254002A1 (en) 2017-09-28 2020-08-13 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
NZ770540A (en) * 2018-06-26 2025-12-19 Nippon Shinyaku Co Ltd Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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
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
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
BR112021010982A2 (pt) 2018-12-07 2021-08-31 Oxford University Innovation Limited Ligantes
EP3954395A4 (en) * 2019-04-08 2023-06-28 National University Corporation Tokyo Medical and Dental University Pharmaceutical composition for muscle disease treatment
WO2021025899A1 (en) 2019-08-02 2021-02-11 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer pharmaceutical compositions
CA3153902A1 (en) 2019-10-16 2021-04-22 Pardis SABETI 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
WO2022213118A1 (en) 2021-03-31 2022-10-06 Entrada Therapeutics, Inc. Cyclic cell penetrating peptides
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
EP4337264A1 (en) 2021-05-10 2024-03-20 Entrada Therapeutics, Inc. Compositions and methods for modulating tissue distribution of intracellular therapeutics
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
MX2024000489A (es) 2021-07-09 2024-04-09 Dyne Therapeutics Inc Complejos dirigidos al musculo y formulaciones para el tratamiento de las distrofinopatías.
US11969475B2 (en) 2021-07-09 2024-04-30 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
US20250066396A1 (en) 2021-12-27 2025-02-27 Nippon Shinyaku Co., Ltd. Method for producing oligonucleic acid compound
EP4493693A1 (en) * 2022-03-17 2025-01-22 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
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
AU5661386A (en) 1985-03-15 1986-10-13 Stirchak, E. Stereoregular polynucleotide-binding polymers
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
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
EP0786522A2 (en) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecules for treatment of stenotic conditions
DK0698092T3 (da) 1993-05-11 2007-11-26 Univ North Carolina Antisenseoligonukleotider, der bekæmper uregelmæssig splejsning,og fremgangsmåder til anvendelse deraf
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
AU2281201A (en) 1999-12-29 2001-07-09 A. James Mixson Histidine copolymer and methods for using same
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
EP1390490B1 (en) 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
DK2206781T3 (en) * 2004-06-28 2016-03-07 Univ Western Australia The antisense oligonucleotides in inducing exon-skipping and methods of use thereof
AU2006213686A1 (en) * 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
KR101789603B1 (ko) 2005-11-10 2017-11-21 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 Ｔｎｆ 수퍼패밀리 수용체에 대한 스플라이스 스위칭올리고머 및 염증성 질환 치료용 약제학적 조성물
SI2024499T1 (en) 2006-05-10 2018-04-30 Sarepta Therapeutics, Inc. Analogues of the oligonucleotide, with cationic links between subunits
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
ES2914775T3 (es) * 2007-10-26 2022-06-16 Academisch Ziekenhuis Leiden Medios y métodos para contrarresta trastornos del músculo
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
CN107353317A (zh) 2010-05-28 2017-11-17 萨勒普塔医疗公司 具有修饰的亚基间键和/或端基的寡核苷酸类似物
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
KR102229650B1 (ko) 2011-05-05 2021-03-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
WO2013033407A2 (en) * 2011-08-30 2013-03-07 The Regents Of The University Of California Identification of small molecules that enhance therapeutic exon skipping
EA031110B1 (ru) * 2012-03-20 2018-11-30 Сарепта Терапьютикс, Инк. Конъюгаты бороновых кислот олигонуклеотидных аналогов

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
JP7008642B2 (ja) 2016-05-24 2022-01-25 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
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