EA201591792A1 - Улучшенные композиции для лечения мышечной дистрофии - Google Patents

Улучшенные композиции для лечения мышечной дистрофии

Info

Publication number
EA201591792A1
EA201591792A1 EA201591792A EA201591792A EA201591792A1 EA 201591792 A1 EA201591792 A1 EA 201591792A1 EA 201591792 A EA201591792 A EA 201591792A EA 201591792 A EA201591792 A EA 201591792A EA 201591792 A1 EA201591792 A1 EA 201591792A1
Authority
EA
Eurasian Patent Office
Prior art keywords
improved compositions
distrophia
muscular
treatment
introducing
Prior art date
Application number
EA201591792A
Other languages
English (en)
Inventor
Эдвард М. Кайе
Original Assignee
Сарепта Терапьютикс, Инк.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Сарепта Терапьютикс, Инк. filed Critical Сарепта Терапьютикс, Инк.
Publication of EA201591792A1 publication Critical patent/EA201591792A1/ru

Links

Classifications

    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • 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
    • 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
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • 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

Abstract

Описаны улучшенные композиции и способы для лечения мышечной дистрофии посредством введения антисмысловой молекулы, способной связываться с выбранным участком-мишенью в гене дистрофина человека для индукции пропуска экзонов.
EA201591792A 2013-03-15 2014-03-14 Улучшенные композиции для лечения мышечной дистрофии EA201591792A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361793463P 2013-03-15 2013-03-15
PCT/US2014/029610 WO2014144978A2 (en) 2013-03-15 2014-03-14 Improved compositions for treating muscular dystrophy

Publications (1)

Publication Number Publication Date
EA201591792A1 true EA201591792A1 (ru) 2016-02-29

Family

ID=50678303

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201591792A EA201591792A1 (ru) 2013-03-15 2014-03-14 Улучшенные композиции для лечения мышечной дистрофии

Country Status (14)

Country Link
US (8) US20140329762A1 (ru)
EP (2) EP3662912A1 (ru)
JP (2) JP2016516066A (ru)
KR (2) KR20150133768A (ru)
CN (2) CN113633787A (ru)
AU (3) AU2014233456B2 (ru)
BR (1) BR112015022998A2 (ru)
CA (1) CA2906812A1 (ru)
EA (1) EA201591792A1 (ru)
HK (1) HK1220154A1 (ru)
IL (3) IL241558B (ru)
MX (2) MX2015013117A (ru)
NZ (2) NZ631289A (ru)
WO (1) WO2014144978A2 (ru)

Families Citing this family (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2206781T3 (pl) 2004-06-28 2016-06-30 Univ Western Australia Antysensowne oligonukleotydy do indukowania pomijania eksonów i sposoby ich zastosowania
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
CN105779453A (zh) 2008-10-24 2016-07-20 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
NZ626359A (en) 2009-11-12 2016-02-26 Univ Western Australia Antisense molecules and methods for treating pathologies
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
EP3998339A1 (en) 2013-03-14 2022-05-18 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
EP3662912A1 (en) 2013-03-15 2020-06-10 Sarepta Therapeutics, Inc. Improved dosages of eteplirsen for treating duchenne muscular dystrophy
BR112017001470A2 (pt) 2014-09-07 2018-02-20 Selecta Biosciences Inc métodos e composições para atenuar as respostas imunes do vetor de transferência anti-viral de terapia genética
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
BR112017024929A2 (pt) 2015-05-19 2018-07-31 Sarepta Therapeutics, Inc. conjugados de oligonucleotídeo-peptídeo
BR112018004970A2 (pt) 2015-09-15 2018-10-09 Nippon Shinyaku Co., Ltd. oligômero antissenso, composição farmacêutica, método para tratamento de distrofia muscular, e, uso do oligômero antissenso ou sal farmaceuticamente aceitável ou hidrato do mesmo.
WO2017059131A1 (en) * 2015-09-30 2017-04-06 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
WO2017062835A2 (en) * 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
WO2017062862A2 (en) 2015-10-09 2017-04-13 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
BR112018011133A2 (pt) * 2015-11-30 2018-11-21 Univ Duke alvos terapêuticos para a correção do gene humano de distrofina por edição de gene e métodos de uso
AU2016372026A1 (en) * 2015-12-15 2018-06-21 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP3464306B1 (en) * 2016-05-24 2024-03-27 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MX2018014472A (es) 2016-05-24 2019-05-23 Sarepta Therapeutics Inc Procesos para preparar oligómeros.
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
AU2017278699A1 (en) * 2016-05-24 2018-11-15 Sarepta Therapeutics, Inc. Pharmaceutical composition comprising Eteplirsen
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
CA3025575A1 (en) * 2016-06-30 2018-01-04 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
CN106581043A (zh) * 2016-10-24 2017-04-26 佳木斯大学 一种适用于杜氏肌营养不良症的药物制剂及其制备方法
CA3044531A1 (en) * 2016-11-28 2018-05-31 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing
LT3554553T (lt) * 2016-12-19 2022-08-25 Sarepta Therapeutics, Inc. Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos
EP4122497B1 (en) 2016-12-19 2024-04-10 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
CA3047010A1 (en) 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
CA3049424A1 (en) * 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
EP3641741B1 (en) 2017-06-19 2024-03-20 University of Maryland, Baltimore Microtubule polymerization inhibitor prodrugs and methods of using the same
US10961537B2 (en) 2017-07-18 2021-03-30 Csl Behring Gene Therapy, Inc. Compositions and methods for treating beta-hemoglobinopathies
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
SG11202001074RA (en) * 2017-08-31 2020-03-30 Sarepta Therapeutics Inc Methods for treating muscular dystrophy
EP3684933A4 (en) * 2017-09-22 2021-06-23 The Regents of the University of Colorado, A Body Corporate OLIGONUCLEOTIDES THIOMORPHOLINO FOR THE TREATMENT OF MUSCLE DYSTROPHY
MA50269A (fr) * 2017-09-22 2020-07-29 Avidity Biosciences Inc Compositions d'acides nucléiques-polypeptides et méthodes d'induction de saut d'exon
EP3687547A1 (en) * 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
MX2020003838A (es) 2017-10-13 2020-08-06 Selecta Biosciences Inc Metodos y composiciones para atenuar las respuestas de inmunoglobulina m del vector de transferencia antiviral.
CN111902148A (zh) 2017-12-06 2020-11-06 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
CN111836893A (zh) * 2018-01-31 2020-10-27 德克萨斯大学系统董事会 用于校正人心肌细胞中肌养蛋白突变的组合物和方法
US20210260206A1 (en) * 2018-03-16 2021-08-26 Sarepta Therapeutics, Inc. Chimeric peptides for antisense deliver
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
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
WO2019241385A2 (en) * 2018-06-13 2019-12-19 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystropy
EP3806868A4 (en) * 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. EXON-SKIPPING OLIGOMERS 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
US11168141B2 (en) 2018-08-02 2021-11-09 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
KR20210081322A (ko) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 디스트로핀병증을 치료하기 위한 그의 용도
KR20210118402A (ko) 2018-12-23 2021-09-30 씨에스엘 베링 엘엘씨 위스콧-알드리치(Wiskott-Aldrich) 증후군에 대한 조혈 줄기 세포-유전자 치료요법
CA3123045A1 (en) 2018-12-23 2020-07-02 Csl Behring L.L.C. Donor t-cells with kill switch
WO2020157760A1 (en) 2019-01-31 2020-08-06 Bar Ilan University Neoantigens created by aberrant-induced splicing and uses thereof in enhancing immunotherapy
BR112021023594A2 (pt) 2019-05-28 2022-02-08 Selecta Biosciences Inc Métodos e composições para resposta imune de vetor de transferência antiviral atenuada
US20220372560A1 (en) * 2019-10-30 2022-11-24 Takeda Pharmaceutical Company Limited Methods for detecting oligonucleotides
AU2020411964A1 (en) * 2019-12-26 2022-06-16 National Center Of Neurology And Psychiatry Antisense nucleic acid that induces skipping of exon 50
EP4121063A1 (en) 2020-03-19 2023-01-25 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
CN115916208A (zh) * 2020-06-17 2023-04-04 康德生物医疗有限公司 用于治疗肌肉萎缩症的方法和组合物
AU2021294317A1 (en) 2020-06-26 2023-02-23 Csl Behring Llc Donor T-cells with kill switch
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
CA3231330A1 (en) 2021-09-16 2023-03-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
WO2023044398A1 (en) * 2021-09-16 2023-03-23 Dyne Therapeutics, Inc. Dosing of muscle targeting complexes for treating dystrophinopathies
WO2023064367A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
WO2023172624A1 (en) 2022-03-09 2023-09-14 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
WO2023178230A1 (en) * 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
CN115820642B (zh) * 2022-11-11 2023-10-10 昆明理工大学 一种用于治疗杜氏肌营养不良症的CRISPR-Cas9系统

Family Cites Families (141)

* 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
US5190931A (en) 1983-10-20 1993-03-02 The Research Foundation Of State University Of New York Regulation of gene expression by employing translational inhibition of MRNA utilizing interfering complementary MRNA
CA1268404A (en) 1985-03-15 1990-05-01 Antivirals Inc. Polynucleotide assay reagent and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
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
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
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
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US20020123476A1 (en) 1991-03-19 2002-09-05 Emanuele R. Martin Therapeutic delivery compositions and methods of use thereof
ES2153379T3 (es) 1992-03-31 2001-03-01 Abbott Lab Procedimiento de reaccion de ligasa en cadena multiplex.
US5869252A (en) 1992-03-31 1999-02-09 Abbott Laboratories Method of multiplex ligase chain reaction
US6806084B1 (en) 1992-06-04 2004-10-19 The Regents Of The University Of California Methods for compositions for in vivo gene delivery
EP1251170A3 (en) 1992-07-17 2002-10-30 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of NF-kappaB dependent animal diseases
US5985558A (en) 1997-04-14 1999-11-16 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the inibition of c-Jun and c-Fos
ES2290951T3 (es) 1993-05-11 2008-02-16 The University Of North Carolina At Chapel Hill Oligonucleotidos antisentido que combaten el corte y empalme aberrante y metodos de uso de los mismos.
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US6391636B1 (en) 1994-05-31 2002-05-21 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of raf gene expression
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
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
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
GB9510718D0 (en) 1995-05-26 1995-07-19 Sod Conseils Rech Applic Antisense oligonucleotides
AU725262B2 (en) 1996-02-14 2000-10-12 Isis Pharmaceuticals, Inc. Sugar-modified gapped oligonucleotides
AU2585197A (en) 1996-03-20 1997-10-10 Regents Of The University Of California, The Antisense approach to gene inhibition
US6153436A (en) 1997-01-10 2000-11-28 The Board Of Trustees Of The University Of Arkansas Method of gene delivery using wildtype adeno associated viral (AAV) vectors with insertions
WO1999042091A2 (en) 1998-02-19 1999-08-26 Massachusetts Institute Of Technology Use of polycations as endosomolytic agents
US20030114401A1 (en) 2001-12-06 2003-06-19 Isis Pharmaceuticals Inc. Antisense modulation of Ship-1 expression
GB9819999D0 (en) 1998-09-14 1998-11-04 Univ London Treatment of cancer
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
CA2350531A1 (en) 1998-11-13 2000-05-25 Smriti Iyengar Method for treating pain
WO2000045167A2 (en) 1999-01-29 2000-08-03 Avi Biopharma, Inc. Non-invasive method for detecting target rna
US20020049173A1 (en) 1999-03-26 2002-04-25 Bennett C. Frank Alteration of cellular behavior by antisense modulation of mRNA processing
JP2000325085A (ja) 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
CA2376284A1 (en) 1999-06-21 2000-12-28 Murdoch Childrens Research Institute A method for the prophylaxis and/or treatment of medical disorders
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
WO2001047496A1 (en) 1999-12-29 2001-07-05 Mixson A James Histidine copolymer and methods for using same
JP2003518946A (ja) 2000-01-04 2003-06-17 エイブイアイ バイオファーマ， インコーポレイテッド アンチセンス抗細菌性細胞分裂組成物、およびその方法
JP2004509604A (ja) 2000-03-28 2004-04-02 アイシス・ファーマシューティカルス・インコーポレーテッド ｍＲＮＡプロセッシングのアンチセンスモジュレーションによる、細胞行動の改変
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
CN100422320C (zh) 2000-04-28 2008-10-01 肖啸 编码抗肌萎缩蛋白小基因的dna序列及其使用方法
CA2407942A1 (en) 2000-05-01 2001-11-08 Hybridon, Inc. Modulation of oligonucleotide cpg-mediated immune stimulation by positional modification of nucleosides
KR20020097241A (ko) 2000-05-04 2002-12-31 에이브이아이 바이오파마 인코포레이티드 스플라이스-영역 안티센스 조성물 및 방법
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
JP4836366B2 (ja) 2000-08-25 2011-12-14 雅文 松尾 デュシェンヌ型筋ジストロフィー治療剤
JP2004513625A (ja) 2000-08-30 2004-05-13 エイブイアイ　バイオファーマ，　インコーポレイテッド オリゴヌクレオチドアナログの分析のための方法
US6559279B1 (en) 2000-09-08 2003-05-06 Isis Pharmaceuticals, Inc. Process for preparing peptide derivatized oligomeric compounds
US20070037165A1 (en) 2000-09-08 2007-02-15 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
WO2002024717A1 (en) 2000-09-20 2002-03-28 Isis Pharmaceuticals, Inc. Antisense modulation of flip-c expression
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US6689615B1 (en) 2000-10-04 2004-02-10 James Murto Methods and devices for processing blood samples
JP3781687B2 (ja) 2001-02-23 2006-05-31 松下電器産業株式会社 遺伝子診断装置及び遺伝子診断方法
US6656732B1 (en) 2001-05-18 2003-12-02 Isis Pharmaceuticals, Inc. Antisense inhibition of src-c expression
US20030224353A1 (en) 2001-10-16 2003-12-04 Stein David A. Antisense antiviral agent and method for treating ssRNA viral infection
JP2006507841A (ja) 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
US7655785B1 (en) 2002-11-14 2010-02-02 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
US7250289B2 (en) 2002-11-20 2007-07-31 Affymetrix, Inc. Methods of genetic analysis of mouse
US7314750B2 (en) 2002-11-20 2008-01-01 Affymetrix, Inc. Addressable oligonucleotide array of the rat genome
CA3001404C (en) 2002-11-25 2020-07-28 Masafumi Matsuo Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
WO2004097017A2 (en) 2003-04-29 2004-11-11 Avi Biopharma, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
CA2526669A1 (en) * 2003-06-02 2004-12-16 Wyeth Use of myostatin (gdf8) inhibitors in conjunction with corticosteroids for treating neuromuscular disorders
DE60319354T2 (de) 2003-07-11 2009-03-26 Lbr Medbiotech B.V. Mannose-6-Phosphat Rezeptor vermittelter Gentransfer zu Muskelzellen
DE602004030583D1 (de) 2003-08-05 2011-01-27 Avi Biopharma Inc Oligonukleotidanalog und verfahren zur behandlung von flavivirusinfektionen
US20050048495A1 (en) 2003-08-29 2005-03-03 Baker Brenda F. Isoform-specific targeting of splice variants
AU2004273828A1 (en) 2003-09-12 2005-03-31 Avi Biopharma, Inc. Compound and method for treating androgen-independent prostate cancer
US20050171044A1 (en) 2003-12-24 2005-08-04 Stein David A. Oligonucleotide compound and method for treating nidovirus infections
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
PL2206781T3 (pl) 2004-06-28 2016-06-30 Univ Western Australia Antysensowne oligonukleotydy do indukowania pomijania eksonów i sposoby ich zastosowania
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
US8129352B2 (en) 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20120122801A1 (en) 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
NO20051491L (no) 2005-03-21 2006-09-22 Norsk Hydro As Fremgangsmate og anordning ved en gravitasjonsseparator.
WO2006112705A2 (en) 2005-04-22 2006-10-26 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secondary rna structure.
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
EP1937312B1 (en) 2005-08-30 2016-06-29 Ionis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
WO2007030691A2 (en) 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
US8501704B2 (en) 2005-11-08 2013-08-06 Sarepta Therapeutics, Inc. Immunosuppression compound and treatment method
AU2006315758A1 (en) 2005-11-10 2007-05-24 Ercole Biotech, Inc. Splice switching oligomers for TNF superfamily receptors and their use in treatment of disease
WO2007133812A2 (en) 2005-12-30 2007-11-22 Philadelphia Health & Education Corporation, D/B/A Drexel University College Of Medicine Improved carriers for delivery of nucleic acid agents to cells and tissues
GB0602767D0 (en) * 2006-02-10 2006-03-22 Vastox Plc Treatment of muscular dystrophy
US8785407B2 (en) 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
US7943762B2 (en) 2006-05-10 2011-05-17 Avi Biopharma, Inc. Oligonucleotide analogs having cationic intersubunit linkages
US20070265215A1 (en) 2006-05-11 2007-11-15 Iversen Patrick L Antisense restenosis composition and method
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
CA2691673A1 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
WO2009054725A2 (en) 2007-10-26 2009-04-30 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
US8076476B2 (en) 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
WO2009101399A1 (en) 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)
ATE529104T1 (de) * 2008-04-09 2011-11-15 Santhera Pharmaceuticals Ch Idebenon zur behandlung von durch muskuläre dystrophie vermittelten atemwegserkrankungen
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
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
CN105779453A (zh) 2008-10-24 2016-07-20 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
EP2344637B2 (en) 2008-10-27 2018-02-28 BioMarin Technologies B.V. Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna
EP2376633A1 (en) 2008-12-17 2011-10-19 AVI BioPharma, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
US20120149756A1 (en) 2009-04-10 2012-06-14 Associatin Institut de Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
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
EP2258863A1 (en) 2009-05-25 2010-12-08 Universita'Degli Studi di Roma "La Sapienza" miRNA biomarkers for the diagnosis of duchenne muscular dystrophy progression, for monitoring therapeutic interventions, and as therapeutics
US9034838B2 (en) 2009-05-25 2015-05-19 Universita Degli Studi Di Roma “La Sapienza” miR-31 in duchenne muscular dystrophy therapy
ITTO20090487A1 (it) 2009-06-26 2010-12-27 Univ Ferrara Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (dmd)
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US20120172415A1 (en) 2009-08-31 2012-07-05 Thomas Voit Exon Skipping Therapy for Functional Amelioration of Semifunctional Dystrophin in Becker and Duchenne Muscular Dystrophy
IT1397011B1 (it) 2009-10-14 2012-12-20 Univ Ferrara Nanoparticella del tipo core-shell idonea per la veicolazione di oligonucleotidi terapeutici in tessuti bersaglio e suo impiego per la preparazione di un medicamento per il trattamento della distrofia muscolare di duchenne.
NZ626359A (en) 2009-11-12 2016-02-26 Univ Western Australia Antisense molecules and methods for treating pathologies
KR101944119B1 (ko) 2009-11-13 2019-01-30 사렙타 쎄러퓨틱스 인코퍼레이티드 안티센스 항바이러스 화합물 및 인플루엔자 바이러스 감염을 치료하는 방법
US9050373B2 (en) 2010-05-13 2015-06-09 The Charlotte-Mecklenburg Hospital Authority Pharmaceutical compositions comprising antisense oligonucleotides and methods of using same
AU2011257980B2 (en) 2010-05-28 2016-06-30 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
JP5950818B2 (ja) 2010-06-28 2016-07-13 正敏 萩原 遺伝性疾患の予防・改善剤
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
DK2623507T3 (da) 2010-09-30 2017-01-02 Nippon Shinyaku Co Ltd Morpholinonukleinsyrederivat
US9078911B2 (en) 2011-02-08 2015-07-14 The Charlotte-Mecklenburg Hospital Authority Antisense oligonucleotides
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
KR102339196B1 (ko) 2011-05-05 2021-12-15 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
EP2750715B1 (en) 2011-08-30 2018-10-31 The Regents of The University of California Identification of small molecules that enhance therapeutic exon skipping
US20140080896A1 (en) 2011-08-30 2014-03-20 The Regents Of The University Of California Identification of small molecules that facilitate therapeutic exon skipping
HUE044369T2 (hu) * 2011-09-05 2019-10-28 Mitsubishi Tanabe Pharma Corp Gyógyászati szer izomsorvadásos laterálszklerózis kezelésére vagy izomsorvadásos laterálszklerózis súlyosbodásának megelõzésére
DK2581448T3 (en) 2011-10-13 2015-04-27 Ass Inst De Myologie Tricyclo-DNA phosphorothioate
JP6141770B2 (ja) 2011-12-28 2017-06-07 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
AU2013235691B2 (en) 2012-03-20 2017-02-02 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
CN110257379B (zh) 2012-07-03 2023-08-11 马林生物科技有限公司 用于治疗肌肉萎缩症患者的寡核苷酸
BR112015014987A2 (pt) 2012-12-20 2017-08-15 Sarepta Therapeutics Inc Aprimoradas composições que pulam éxon para o tratamento da distrofia muscular
KR20230074606A (ko) 2013-03-14 2023-05-30 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
EP3998339A1 (en) 2013-03-14 2022-05-18 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
EP3662912A1 (en) 2013-03-15 2020-06-10 Sarepta Therapeutics, Inc. Improved dosages of eteplirsen for treating duchenne muscular dystrophy
CN109652385A (zh) 2013-04-20 2019-04-19 全国儿童医院研究所 外显子2靶向U7snRNA多核苷酸构建体的重组腺相关病毒递送
WO2017059131A1 (en) 2015-09-30 2017-04-06 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy

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