[go: up one dir, main page]

MX2019008209A - Composiciones para el salto del exon para el tratamiento de distrofia muscular. - Google Patents

Composiciones para el salto del exon para el tratamiento de distrofia muscular.

Info

Publication number
MX2019008209A
MX2019008209A MX2019008209A MX2019008209A MX2019008209A MX 2019008209 A MX2019008209 A MX 2019008209A MX 2019008209 A MX2019008209 A MX 2019008209A MX 2019008209 A MX2019008209 A MX 2019008209A MX 2019008209 A MX2019008209 A MX 2019008209A
Authority
MX
Mexico
Prior art keywords
muscular dystrophy
exon skipping
treating muscular
skipping compositions
compositions
Prior art date
Application number
MX2019008209A
Other languages
English (en)
Inventor
Joseph Schnell Fred
Elizabeth Frank Diane
K Bestwick Richard
Original Assignee
Sarepta Therapeutics Inc
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 Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of MX2019008209A publication Critical patent/MX2019008209A/es

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K47/56—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 organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—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 organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—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 organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • A61K47/62—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
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/32—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
    • 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
    • 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
    • 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
    • C12N2310/35—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
    • 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
    • C12N2310/35—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
    • 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
    • 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
    • C12N2320/33—Alteration of splicing
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Polymers With Sulfur, Phosphorus Or Metals In The Main Chain (AREA)

Abstract

Se describen moléculas antisentido capaces de unirse a un sitio objetivo seleccionado en el gen de la distrofina humana para inducir el salto del exón 44.
MX2019008209A 2013-03-14 2015-09-10 Composiciones para el salto del exon para el tratamiento de distrofia muscular. MX2019008209A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201361784547P 2013-03-14 2013-03-14

Publications (1)

Publication Number Publication Date
MX2019008209A true MX2019008209A (es) 2019-09-13

Family

ID=50694004

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2015012148A MX366485B (es) 2013-03-14 2014-03-14 Composiciones para el salto de exón para tratamiento de distrofia muscular.
MX2019008209A MX2019008209A (es) 2013-03-14 2015-09-10 Composiciones para el salto del exon para el tratamiento de distrofia muscular.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2015012148A MX366485B (es) 2013-03-14 2014-03-14 Composiciones para el salto de exón para tratamiento de distrofia muscular.

Country Status (15)

Country Link
US (11) US9217148B2 (es)
EP (3) EP2970963B1 (es)
JP (5) JP6449231B2 (es)
KR (7) KR20230116945A (es)
CN (3) CN105378081B (es)
AU (4) AU2014236140B2 (es)
BR (2) BR122020016865B1 (es)
CA (1) CA2906209A1 (es)
EA (2) EA035882B1 (es)
ES (1) ES2762881T3 (es)
IL (3) IL280443B (es)
MX (2) MX366485B (es)
NZ (4) NZ731587A (es)
SA (1) SA515361125B1 (es)
WO (1) WO2014153220A2 (es)

Families Citing this family (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4272748A3 (en) 2004-06-28 2024-03-27 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
SI3133160T1 (sl) 2008-10-24 2019-05-31 Sarepta Therapeutics, Inc. Sestavki, ki preskakujejo ekson za DMD
SMT201800579T1 (it) 2009-11-12 2019-01-11 Univ Western Australia Molecole antisenso e metodi per trattare patologie
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
CN105378081B (zh) 2013-03-14 2019-06-14 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
HK1220154A1 (zh) 2013-03-15 2017-04-28 Sarepta Therapeutics, Inc. 改进的用於治疗肌营养不良的组合物
ES2797679T3 (es) * 2013-12-02 2020-12-03 Ionis Pharmaceuticals Inc Compuestos antisentido y usos de los mismos
US20180216111A1 (en) * 2015-02-27 2018-08-02 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
WO2016149455A2 (en) * 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
MA45819A (fr) 2015-10-09 2018-08-15 Sarepta Therapeutics Inc Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés
MA46427A (fr) 2015-10-09 2018-08-15 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
JOP20200228A1 (ar) 2015-12-21 2017-06-16 Novartis Ag تركيبات وطرق لخفض تعبير البروتين tau
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
JP7033547B2 (ja) 2016-04-18 2022-03-10 サレプタ セラピューティクス， インコーポレイテッド 酸性アルファ－グルコシダーゼ遺伝子に関連する疾患を処置するためのアンチセンスオリゴマーおよびそれを用いる方法
AU2017270975B2 (en) 2016-05-24 2021-04-15 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
HRP20241428T1 (hr) * 2016-05-24 2025-01-03 Sarepta Therapeutics, Inc. Postupci za pripremu oligomera
AU2017270598B2 (en) * 2016-05-24 2022-12-01 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
RS65504B1 (sr) * 2016-05-24 2024-06-28 Sarepta Therapeutics Inc Procesi za pripremu fosfordiamidat morfolino oligomera
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
AU2017382773B2 (en) 2016-12-19 2025-01-30 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
LT4122497T (lt) * 2016-12-19 2024-06-10 Sarepta Therapeutics, Inc. Egzonus praleidžiantys oligomerų konjugatai, skirti raumenų distrofijai gydyti
KR102552428B1 (ko) 2016-12-19 2023-07-06 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
SG10202107429WA (en) 2017-01-06 2021-08-30 Avidity Biosciences Inc 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
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
JP7394753B2 (ja) 2017-10-18 2023-12-08 サレプタ セラピューティクス， インコーポレイテッド アンチセンスオリゴマー化合物
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
US10758629B2 (en) 2018-05-29 2020-09-01 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
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
WO2020028864A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral 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
IT201800009682A1 (it) * 2018-10-22 2020-04-22 Ice Spa Coniugati di acidi biliari e loro derivati per la veicolazione di molecole attive
US12398140B2 (en) 2019-02-13 2025-08-26 Ptc Therapeutics, Inc. Substituted pyrrolo [2,3-d]pyrimidines for treating familial dysautonomia
AU2020222881C1 (en) 2019-02-13 2026-01-22 Ptc Therapeutics, Inc. Thioeno(3,2-b) pyridin-7-amine compounds for treating familial dysautonomia
US12497615B2 (en) 2019-04-25 2025-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
AU2020324957A1 (en) * 2019-08-02 2022-03-03 Research Institute At Nationwide Children's Hospital Exon 44-targeted nucleic acids and recombinant adeno-associated virus comprising said nucleic acids for treatment of dystrophin-based myopathies
TW202136511A (zh) * 2019-12-19 2021-10-01 日商日本新藥股份有限公司 能進行外顯子跳讀之反義核酸
AU2021237465A1 (en) 2020-03-19 2022-10-13 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
KR20230079183A (ko) * 2020-09-30 2023-06-05 바이오마린 테크놀로지스 비.브이. 디스트로핀 유전자의 엑손 51을 표적화하는 안티센스 올리고뉴클레오타이드
WO2023283623A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11771776B2 (en) * 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
CN120648680A (zh) * 2024-03-15 2025-09-16 迦进生物医药(上海)有限公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的药物

Family Cites Families (149)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6683A (en) 1849-08-28 And john w
US173A (en) 1837-04-20 Improvement in the manufacture of hat-bodies
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
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
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
DE3650349T2 (de) 1985-03-15 1995-12-14 Antivirals Inc Immunotestmittel für polynukleotid und verfahren.
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
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
US5508337A (en) 1992-02-11 1996-04-16 Bayer Aktiengesellschaft Powder coating compositions, a process for their preparation, and their use for the coating of heat resistant substrates
US5869252A (en) 1992-03-31 1999-02-09 Abbott Laboratories Method of multiplex ligase chain reaction
EP0633944B1 (en) 1992-03-31 2000-11-08 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
CA2140343A1 (en) 1992-07-17 1994-02-03 Sean M. Sullivan Method and reagent for treatment of 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
DK0698092T3 (da) 1993-05-11 2007-11-26 Univ North Carolina Antisenseoligonukleotider, der bekæmper uregelmæssig splejsning,og fremgangsmåder til anvendelse deraf
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
EP0882061B1 (en) 1996-02-14 2004-05-19 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
US6683173B2 (en) 1998-04-03 2004-01-27 Epoch Biosciences, Inc. Tm leveling methods
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
ES2194536T3 (es) 1998-11-13 2003-11-16 Lilly Co Eli Combinacion de duloxetina con farmacos antiinflamatorios no esteroideos.
KR20010102992A (ko) 1999-01-29 2001-11-17 추후보정 표적 ｒｎａ를 검출하는 비-침입적 방법
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
AU768904B2 (en) 1999-06-21 2004-01-08 Murdoch Childrens Research Institute, The 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
US7163695B2 (en) 1999-12-29 2007-01-16 Mixson A James Histidine copolymer and methods for using same
US7049431B2 (en) 2000-01-04 2006-05-23 Avi Biopharma, Inc. Antisense antibacterial cell division composition and method
AU4036600A (en) 2000-03-28 2001-10-08 Isis Pharmaceuticals Inc Alteration of cellular behavior by antisense modulation of mrna processing
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
AU2001261063A1 (en) 2000-04-28 2001-11-12 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
PT1278761E (pt) 2000-05-01 2005-08-31 Hybridon Inc Modulacao da estimulacao imunitaria mediada por oligonucleotidos de cpg, por modificacao posicional de nucleosidos
EP1282699B1 (en) 2000-05-04 2012-11-21 Sarepta Therapeutics, Inc. Splice-region antisense composition and method
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
US20070037165A1 (en) 2000-09-08 2007-02-15 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
US6559279B1 (en) 2000-09-08 2003-05-06 Isis Pharmaceuticals, Inc. Process for preparing peptide derivatized oligomeric compounds
EP1319014A1 (en) 2000-09-20 2003-06-18 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
EP2017338A1 (en) 2001-05-24 2009-01-21 Genzyme Corporation Muscle-specific expression vectors
US20030224353A1 (en) 2001-10-16 2003-12-04 Stein David A. Antisense antiviral agent and method for treating ssRNA viral infection
US7655785B1 (en) 2002-11-14 2010-02-02 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
EP1560931B1 (en) 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
US7314750B2 (en) 2002-11-20 2008-01-01 Affymetrix, Inc. Addressable oligonucleotide array of the rat genome
US7250289B2 (en) 2002-11-20 2007-07-31 Affymetrix, Inc. Methods of genetic analysis of mouse
AU2003284638A1 (en) 2002-11-25 2004-06-18 Nonprofit Organization Translational Research Organization Of Duchenne Muscular Dystrophy ENA NUCLEIC ACID DRUGS MODIFYING SPLICING IN mRNA PRECURSOR
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
ES2500921T3 (es) 2003-04-29 2014-10-01 Sarepta Therapeutics, Inc. Composiciones para potenciar el transporte y la eficacia antisentido de análogos de ácidos nucleicos en células
ATE387217T1 (de) 2003-07-11 2008-03-15 Lbr Medbiotech B V Mannose-6-phosphat rezeptor vermittelter gentransfer zu muskelzellen
KR100943473B1 (ko) 2003-08-05 2010-02-19 에이브이아이 바이오파마 인코포레이티드 올리고뉴클레오티드 유사체 및 플라비바이러스 감염을 치료하는 방법
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
EP4272748A3 (en) 2004-06-28 2024-03-27 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
FR2873294B1 (fr) 2004-07-26 2008-05-09 Greenpharma Sa Sa Association de medicaments
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
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
NZ563206A (en) 2005-04-22 2009-09-25 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mRNA by interfering with the binding or 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
US8501703B2 (en) 2005-08-30 2013-08-06 Isis 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
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
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
EP2735568B8 (en) 2006-05-10 2017-09-27 Sarepta Therapeutics, Inc. Oligonucleotide analogs having cationic intersubunit linkages
US8785407B2 (en) 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
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
BE1017460A6 (nl) 2007-02-09 2008-10-07 Leo Vermeulen Consulting Lvc Lenticulair folie.
US8251804B2 (en) 2007-04-23 2012-08-28 Wms Gaming Inc. Gaming system having progressive jackpots flexibly linked with common progressive pool
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
AU2008271050B2 (en) 2007-06-29 2014-11-06 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
EP2203173B1 (en) 2007-10-26 2015-12-23 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
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
WO2009101399A1 (en) 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
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
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
SI3133160T1 (sl) 2008-10-24 2019-05-31 Sarepta Therapeutics, Inc. Sestavki, ki preskakujejo ekson za DMD
PT2607484E (pt) 2008-10-27 2016-03-09 Academisch Ziekenhuis Leiden Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne
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
EP2421971B1 (en) 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
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.
SMT201800579T1 (it) 2009-11-12 2019-01-11 Univ Western Australia Molecole antisenso e metodi per trattare patologie
AU2010319314C1 (en) 2009-11-13 2016-09-01 Sarepta Therapeutics, Inc. Antisense antiviral compound and method for treating influenza viral infection
WO2011064552A1 (en) * 2009-11-30 2011-06-03 Isis Innovation Limited Conjugates for delivery of biologically active compounds
US9050373B2 (en) 2010-05-13 2015-06-09 The Charlotte-Mecklenburg Hospital Authority Pharmaceutical compositions comprising antisense oligonucleotides and methods of using same
WO2011150408A2 (en) * 2010-05-28 2011-12-01 Avi Biopharma, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
CN103096928A (zh) 2010-06-28 2013-05-08 萩原正敏 遗传性疾病的预防/改善剂
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
AU2011367230B2 (en) * 2011-05-05 2017-08-10 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
CN102856180B (zh) 2011-06-30 2016-05-25 中国科学院微电子研究所 一种半导体器件的替代栅集成方法
WO2013033407A2 (en) 2011-08-30 2013-03-07 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
ES2535654T3 (es) 2011-10-13 2015-05-13 Association Institut De Myologie ADN triciclo-fosforotioato
CN108486116A (zh) 2011-12-28 2018-09-04 日本新药株式会社 反义核酸
ES2907250T3 (es) 2012-01-27 2022-04-22 Biomarin Tech Bv Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker
CA2868174A1 (en) * 2012-03-20 2013-09-26 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
EP3608407A1 (en) 2012-07-03 2020-02-12 BioMarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
WO2014100714A1 (en) 2012-12-20 2014-06-26 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
CN105378081B (zh) 2013-03-14 2019-06-14 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
EP2970964B8 (en) 2013-03-14 2019-03-06 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
HK1220154A1 (zh) 2013-03-15 2017-04-28 Sarepta Therapeutics, Inc. 改进的用於治疗肌营养不良的组合物
AU2014253730B2 (en) 2013-04-20 2018-09-13 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted U7snRNA polynucleotide constructs
US9505058B2 (en) 2014-05-16 2016-11-29 Xerox Corporation Stabilized metallic nanoparticles for 3D printing
CN108738310A (zh) 2015-09-30 2018-11-02 萨勒普塔医疗公司 用于治疗肌营养不良的方法

Also Published As

Publication number Publication date
MX366485B (es) 2019-07-10
AU2019283961B2 (en) 2022-03-31
BR112015023001A2 (pt) 2017-11-14
US9217148B2 (en) 2015-12-22
EP3633035A1 (en) 2020-04-08
EA202090946A2 (ru) 2021-04-30
HK1219755A1 (en) 2017-04-13
US20180016574A1 (en) 2018-01-18
CN105378081A (zh) 2016-03-02
KR102258326B1 (ko) 2021-06-02
KR20210063456A (ko) 2021-06-01
KR102521608B1 (ko) 2023-04-14
EA202090946A3 (ru) 2021-07-30
NZ775701A (en) 2022-08-26
AU2022202393B2 (en) 2025-02-27
JP2016517279A (ja) 2016-06-16
AU2025200952A1 (en) 2025-03-06
IL293975A (en) 2022-08-01
NZ631245A (en) 2017-09-29
BR112015023001B8 (pt) 2022-08-09
US20190177724A1 (en) 2019-06-13
US10907154B2 (en) 2021-02-02
IL240984A0 (en) 2015-11-30
JP2019150053A (ja) 2019-09-12
IL293975B1 (en) 2025-09-01
KR20230051639A (ko) 2023-04-18
KR20150133751A (ko) 2015-11-30
KR102558958B1 (ko) 2023-07-25
EA201591767A8 (ru) 2018-04-30
US20220056442A1 (en) 2022-02-24
IL280443B (en) 2022-07-01
EP3998339A1 (en) 2022-05-18
JP2018126160A (ja) 2018-08-16
US20190330624A1 (en) 2019-10-31
KR20230116945A (ko) 2023-08-04
EP2970963B1 (en) 2019-10-23
US20210180064A1 (en) 2021-06-17
US20140329881A1 (en) 2014-11-06
SA515361125B1 (ar) 2020-09-03
IL280443A (en) 2021-03-01
ES2762881T3 (es) 2020-05-26
IL240984B (en) 2021-02-28
BR112015023001A8 (pt) 2018-01-23
CN117844807A (zh) 2024-04-09
EA201591767A1 (ru) 2016-02-29
JP2024060002A (ja) 2024-05-01
AU2022202393A1 (en) 2022-05-05
MX2015012148A (es) 2016-06-02
CN110218727A (zh) 2019-09-10
KR20240034881A (ko) 2024-03-14
AU2014236140A1 (en) 2015-10-01
KR20220054720A (ko) 2022-05-03
JP2021166541A (ja) 2021-10-21
US20250236869A1 (en) 2025-07-24
JP6760992B2 (ja) 2020-09-23
US20160298111A1 (en) 2016-10-13
EP2970963A2 (en) 2016-01-20
US20250145991A1 (en) 2025-05-08
IL293975B2 (en) 2026-01-01
WO2014153220A2 (en) 2014-09-25
NZ731587A (en) 2021-07-30
WO2014153220A3 (en) 2014-12-04
AU2019283961A1 (en) 2020-01-23
US20140323544A1 (en) 2014-10-30
EA035882B1 (ru) 2020-08-27
KR102390965B1 (ko) 2022-04-26
BR122020016865B1 (pt) 2022-12-27
NZ790263A (en) 2023-07-28
CA2906209A1 (en) 2014-09-25
KR20250047401A (ko) 2025-04-03
CN105378081B (zh) 2019-06-14
JP6449231B2 (ja) 2019-01-09
US20170369876A1 (en) 2017-12-28
AU2014236140B2 (en) 2019-10-03
BR112015023001B1 (pt) 2022-07-19
US11932851B2 (en) 2024-03-19

Similar Documents

Publication Publication Date Title
MX2019008209A (es) Composiciones para el salto del exon para el tratamiento de distrofia muscular.
CO2019007398A2 (es) Conjugados de oligómeros de omisión de exón para distrofia muscular
CO2019007397A2 (es) Conjugados de oligómeros de omisión de exón para distrofia muscular
CO2019007399A2 (es) Conjugados de oligómeros de omisión de exón para distrofia muscular
MX2015013117A (es) Composiciones mejoradas para tratar distrofia muscular.
CY1121876T1 (el) Συνθεσεις παρακαμψης εξωνιου για τη θεραπεια της μυϊκης δυστροφiας
CO2018014029A2 (es) Oligómeros de omisión de exon para distrofia muscular
BR112015014987A2 (pt) Aprimoradas composições que pulam éxon para o tratamento da distrofia muscular
MX386935B (es) Profármacos de fumaratos y su uso en el tratamiento de diferentes enfermedades.
BR112016025427A2 (pt) processos de preparação de um inibidor de jak1 e formas do mesmo
ECSP17064695A (es) Compuestos y composiciones de alfa-cinamida como inhibidores de hdac8
CR20160321A (es) Modulares de tetrahidropiridopirazinas de gpr6
ECSP14017269A (es) Compuestos para tratar la atrofia muscular espinal
BR112018011633A2 (pt) inibidores de aza-benzimidazol de pad4
CL2014002661A1 (es) Compuestos derivados de tienopirimidina o sus sales, como inhibidores de la alk quinasa; su proceso de preparacion; composicion farmaceutica que los comprende, util para el tratamiento del cancer.
PT2859896T (pt) Composições farmacêuticas para o tratamento de distúrbios musculares
BR112017009289A2 (pt) métodos de administrar composições de amantadina
CL2017000312A1 (es) Elección de microarn como diana para trastornos metabólicos
MX2015009277A (es) Formas de dosificacion y usos terapauticos de l-4-cloro quinurenina.
CY1119470T1 (el) Γαλακταγωγες συνθεσεις με βαση φωσφατιδυλοσερινη
CO6990150A1 (es) Combinación de compuestos derivados del ácido gálico para el tratamiento del cáncer
FR3027522B1 (fr) Composition poreuse chargee en principe actif
CO7071112A2 (es) Planta de tratamiento para el tratamiento de aguas residuales
FR3046029B1 (fr) Composition fertilisante
ITUB20156862A1 (it) Composizioni per il trattamento della psoriasi.