EA201591178A1 - Улучшенные композиции для пропускания экзона для лечения мышечной дистрофии - Google Patents

Улучшенные композиции для пропускания экзона для лечения мышечной дистрофии

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Publication number
EA201591178A1
EA201591178A1 EA201591178A EA201591178A EA201591178A1 EA 201591178 A1 EA201591178 A1 EA 201591178A1 EA 201591178 A EA201591178 A EA 201591178A EA 201591178 A EA201591178 A EA 201591178A EA 201591178 A1 EA201591178 A1 EA 201591178A1
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EA
Eurasian Patent Office
Prior art keywords
distrophia
improved compositions
treating muscle
extract passing
extract
Prior art date
Application number
EA201591178A
Other languages
English (en)
Russian (ru)
Inventor
Ричард К. Бестуик
Дайан Элизабет Франк
Original Assignee
Сарепта Терапьютикс, Инк.
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Application filed by Сарепта Терапьютикс, Инк. filed Critical Сарепта Терапьютикс, Инк.
Publication of EA201591178A1 publication Critical patent/EA201591178A1/ru

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    • C12N15/113Non-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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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2320/33Alteration of splicing

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EA201591178A 2012-12-20 2013-12-20 Улучшенные композиции для пропускания экзона для лечения мышечной дистрофии EA201591178A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261739968P 2012-12-20 2012-12-20
PCT/US2013/077216 WO2014100714A1 (en) 2012-12-20 2013-12-20 Improved exon skipping compositions for treating muscular dystrophy

Publications (1)

Publication Number Publication Date
EA201591178A1 true EA201591178A1 (ru) 2015-11-30

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EA201591178A EA201591178A1 (ru) 2012-12-20 2013-12-20 Улучшенные композиции для пропускания экзона для лечения мышечной дистрофии

Country Status (13)

Country Link
US (5) US20150361428A1 (https=)
EP (2) EP3885439A1 (https=)
JP (3) JP2016502858A (https=)
KR (2) KR20150099804A (https=)
CN (2) CN111440796A (https=)
AU (3) AU2013364158A1 (https=)
BR (1) BR112015014987A2 (https=)
CA (1) CA2894899A1 (https=)
EA (1) EA201591178A1 (https=)
HK (1) HK1216902A1 (https=)
IL (1) IL239491A0 (https=)
MX (2) MX2015008035A (https=)
WO (1) WO2014100714A1 (https=)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2933332A1 (en) 2004-06-28 2015-10-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
TR201816523T4 (tr) 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
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
US20140315977A1 (en) * 2013-03-14 2014-10-23 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
CN110218727A (zh) 2013-03-14 2019-09-10 萨勒普塔医疗公司 用于治疗肌营养不良的外显子跳跃组合物
US20140329762A1 (en) 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
PT3283500T (pt) * 2015-04-08 2021-01-28 Univ Chicago Composições e métodos para corrigir distrofia muscular das cinturas tipo 2c com utilização de salto do exão
EP3858993A1 (en) 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
PH12018501207B1 (en) 2015-12-21 2024-02-23 Novartis Ag Compositions and methods for decreasing tau expression
WO2017136435A1 (en) 2016-02-01 2017-08-10 The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health Compounds for modulating fc-epsilon-ri-beta expression and uses thereof
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
JP2019525742A (ja) 2016-06-30 2019-09-12 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマー
EP3481432A4 (en) 2016-07-05 2020-05-06 The U.S.A. as represented by the Secretary, Department of Health and Human Services Diagnosing col6-related disorders and methods for treating same
US10898566B2 (en) 2016-09-19 2021-01-26 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Zika virus vaccines
KR20240006023A (ko) 2016-12-19 2024-01-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
KR102810425B1 (ko) 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
PT3554552T (pt) 2016-12-19 2022-10-03 Sarepta Therapeutics Inc Conjugados de oligómero de skipping de exão para distrofia muscular
MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
WO2018152526A1 (en) 2017-02-20 2018-08-23 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Zika virus vaccines
GB201711809D0 (en) * 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
EA201991450A1 (ru) * 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
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
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
IL318474A (en) * 2018-12-13 2025-03-01 Sarepta Therapeutics Inc Axon-skipping oligomer complexes for muscular dystrophy
CN118516348B (zh) * 2024-07-23 2025-01-17 深川生物科技(山东)股份公司 单链核酸或双链核酸及在制备治疗杜氏肌营养不良的药物中的应用

Family Cites Families (42)

* 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
US3426011A (en) 1967-02-13 1969-02-04 Corn Products Co Cyclodextrins with anionic properties
US3453257A (en) 1967-02-13 1969-07-01 Corn Products Co Cyclodextrin with cationic 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
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
WO1986005519A1 (en) 1985-03-15 1986-09-25 James Summerton Polynucleotide assay reagent and method
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library 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
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral 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
EP0786522A2 (en) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecules for treatment of stenotic conditions
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
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
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
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
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
EP1390490B1 (en) 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
EP2933332A1 (en) 2004-06-28 2015-10-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2007058894A2 (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
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
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
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
US8084601B2 (en) * 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
BR122020021379B1 (pt) * 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
TR201816523T4 (tr) * 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
KR20200133284A (ko) 2010-05-28 2020-11-26 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
CA3092114A1 (en) 2011-05-05 2012-11-08 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates

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JP2016502858A (ja) 2016-02-01
CA2894899A1 (en) 2014-06-26
BR112015014987A2 (pt) 2017-08-15
US20200339985A1 (en) 2020-10-29
EP3885439A1 (en) 2021-09-29
JP2019050834A (ja) 2019-04-04
MX2019008164A (es) 2019-09-13
JP2018110601A (ja) 2018-07-19
US20220259592A1 (en) 2022-08-18
IL239491A0 (en) 2015-08-31
AU2013364158A1 (en) 2015-07-09
US20170369875A1 (en) 2017-12-28
MX2015008035A (es) 2016-03-17
CN106414739A (zh) 2017-02-15
CN111440796A (zh) 2020-07-24
AU2019253778A1 (en) 2019-11-07
KR20150099804A (ko) 2015-09-01
US20230104708A1 (en) 2023-04-06
HK1216902A1 (zh) 2016-12-09
AU2022201628A1 (en) 2022-03-31
WO2014100714A1 (en) 2014-06-26
EP2935584A1 (en) 2015-10-28
KR20200143739A (ko) 2020-12-24
US20150361428A1 (en) 2015-12-17

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