MA45819A - Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés - Google Patents

Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés

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Publication number
MA45819A
MA45819A MA045819A MA45819A MA45819A MA 45819 A MA45819 A MA 45819A MA 045819 A MA045819 A MA 045819A MA 45819 A MA45819 A MA 45819A MA 45819 A MA45819 A MA 45819A
Authority
MA
Morocco
Prior art keywords
related disorders
methods
compositions
muscle dystrophy
treating duchenne
Prior art date
Application number
MA045819A
Other languages
English (en)
Inventor
George Dickson
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 MA45819A publication Critical patent/MA45819A/fr

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1774—Immunoglobulin superfamily (e.g. CD2, CD4, CD8, ICAM molecules, B7 molecules, Fc-receptors, MHC-molecules)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
    • C07F9/65586—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system at least one of the hetero rings does not contain nitrogen as ring hetero atom
    • 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
    • C12N2310/35—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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Psychology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La présente invention concerne des compositions et des procédés de traitement de la dystrophie musculaire de duchenne et de troubles associés. Des oligomères antisens modifiés sont décrits pour le traitement de la dystrophie musculaire de duchenne et de troubles associés.
MA045819A 2015-10-09 2016-10-07 Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés MA45819A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562239812P 2015-10-09 2015-10-09

Publications (1)

Publication Number Publication Date
MA45819A true MA45819A (fr) 2018-08-15

Family

ID=58488705

Family Applications (1)

Application Number Title Priority Date Filing Date
MA045819A MA45819A (fr) 2015-10-09 2016-10-07 Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés

Country Status (9)

Country Link
US (1) US20190177723A1 (fr)
EP (2) EP3359668A4 (fr)
JP (2) JP2018530560A (fr)
CN (1) CN108699555A (fr)
BR (1) BR112018007066A2 (fr)
EA (1) EA201890908A1 (fr)
IL (2) IL258069A (fr)
MA (1) MA45819A (fr)
WO (1) WO2017062835A2 (fr)

Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
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
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
US20190262375A1 (en) * 2016-06-30 2019-08-29 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
CN109757108A (zh) * 2016-07-05 2019-05-14 比奥马林技术公司 具有治疗遗传疾病的改善特征的包含双环支架部分的前体mRNA剪接转换或调节寡核苷酸
KR102646318B1 (ko) 2016-12-19 2024-03-12 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
HRP20240705T1 (hr) * 2016-12-19 2024-08-30 Sarepta Therapeutics, Inc. Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju
AR112538A1 (es) * 2017-08-31 2019-11-06 Sarepta Therapeutics Inc Métodos para el tratamiento de distrofia muscular
EA201991450A1 (ru) * 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
TWI812647B (zh) * 2017-09-25 2023-08-21 美商薩羅塔治療公司 經由速流合成以製備磷醯二胺嗎啉代寡聚物之製程
US20200384084A1 (en) 2017-12-01 2020-12-10 Merus N.V. Use of bispecific antibody and il-15 for combination therapy
BR112020015617A2 (pt) * 2018-01-31 2021-01-05 The Board Of Regents Of The University Of Texas System Composições e métodos para correção de mutações da distrofina em cardiomiócitos humanos
BR112020020670A2 (pt) 2018-04-12 2021-03-02 Wave Life Sciences Ltd. composição de oligonucleotídeo, composição farmacêutica, método para alterar o splicing de uma transcrição alvo, método para tratar distrofia muscular, método para preparar um oligonucleotídeo ou uma composição de oligonucleotídeo do mesmo e oligonucleotídeo
EP3784248A4 (fr) * 2018-04-26 2022-08-10 Sarepta Therapeutics, Inc. Oligomères induisant le saut d'exon et conjugués d'oligomères pour la dystrophie musculaire
TWI844541B (zh) * 2018-05-11 2024-06-11 新加坡商波濤生命科學有限公司 寡核苷酸組成物及其使用方法
US10765760B2 (en) * 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3806868A4 (fr) * 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. Oligomères induisant un saut d'exon pour la dystrophie musculaire
WO2019241385A2 (fr) * 2018-06-13 2019-12-19 Sarepta Therapeutics, Inc. Oligomères induisant un saut d'exon pour la dystrophie musculaire
WO2019241470A2 (fr) * 2018-06-14 2019-12-19 Sarepta Therapeutics, Inc. Oligomères induisant le saut d'exon et conjugués d'oligomères pour la dystrophie musculaire
TW202449155A (zh) * 2018-07-27 2024-12-16 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
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
AU2019316103B2 (en) 2018-08-02 2026-02-19 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
WO2020028832A1 (fr) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs utilisations pour le traitement de dystrophinopathies
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
EP3874044A1 (fr) * 2018-11-02 2021-09-08 BioMarin Technologies B.V. Oligonucléotides antisens bispécifiques pour le saut d'exon de la dystrophine
AU2019392928A1 (en) * 2018-12-06 2021-06-17 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
EP3890783A1 (fr) 2018-12-07 2021-10-13 Oxford University Innovation Limited Lieurs
SG11202104960PA (en) * 2018-12-13 2021-06-29 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
GB201821269D0 (en) * 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
WO2020209285A1 (fr) * 2019-04-08 2020-10-15 国立大学法人東京医科歯科大学 Composition pharmaceutique pour traitement des maladies musculaires
EP3959319A4 (fr) 2019-04-25 2023-06-07 Avidity Biosciences, Inc. Compositions d'acide nucléique et méthodes de saut multi-exon
BR112021022208A2 (pt) * 2019-05-06 2022-01-11 Antisense Therapeutics Ltd Métodos para tratamento de distrofia muscular usando oligonucleotídeos inibidores para cd49d
US20220282247A1 (en) * 2019-08-02 2022-09-08 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
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
CN115348883B (zh) * 2019-11-27 2024-11-15 诺华股份有限公司 用于治疗杜兴氏肌营养不良症的化合物和方法
AU2020411964A1 (en) * 2019-12-26 2022-06-16 National Center Of Neurology And Psychiatry Antisense nucleic acid that induces skipping of exon 50
UY39444A (es) * 2020-09-30 2022-04-29 Biomarin Tech Bv Oligonucleótido
EP4299743A4 (fr) * 2021-02-26 2025-01-29 KNC Laboratories Co., Ltd. Médicament à base d'acide nucléique exprimant un variant d'épissage de la myostatine
WO2022192749A2 (fr) * 2021-03-12 2022-09-15 Pepgen Inc. Méthodes de traitement de la dystrophie musculaire de duchenne à l'aide de conjugués peptide-oligonucléotide
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス，インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
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
JP2025509438A (ja) * 2022-03-17 2025-04-11 サレプタ セラピューティクス， インコーポレイテッド ホスホロジアミデートモルホリノオリゴマーコンジュゲート
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
US20250288641A1 (en) * 2022-04-27 2025-09-18 The Curators Of The University Of Missouri Micro-dystrophin for heart protection
WO2025072246A1 (fr) * 2023-09-26 2025-04-03 Entrada Therapeutics, Inc. Composés et procédés pour sauter l'exon 50 dans le cadre de la dystrophie musculaire de duchenne
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
WO2025262186A2 (fr) * 2024-06-20 2025-12-26 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Oligonucléotides thérapeutiques pour le traitement de maladies associées ou causées par une mutation contre-sens, non-sens ou indel dans le cadre

Family Cites Families (118)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4534899A (en) 1981-07-20 1985-08-13 Lipid Specialties, Inc. Synthetic phospholipid compounds
US4426330A (en) 1981-07-20 1984-01-17 Lipid Specialties, Inc. Synthetic phospholipid compounds
AU5661386A (en) 1985-03-15 1986-10-13 Stirchak, E. Stereoregular polynucleotide-binding polymers
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
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
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
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5354844A (en) 1989-03-16 1994-10-11 Boehringer Ingelheim International Gmbh Protein-polycation conjugates
US5108921A (en) 1989-04-03 1992-04-28 Purdue Research Foundation Method for enhanced transmembrane transport of exogenous molecules
US5227170A (en) 1989-06-22 1993-07-13 Vestar, Inc. Encapsulation process
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5356633A (en) 1989-10-20 1994-10-18 Liposome Technology, Inc. Method of treatment of inflamed tissues
US5527528A (en) 1989-10-20 1996-06-18 Sequus Pharmaceuticals, Inc. Solid-tumor treatment method
US5469854A (en) 1989-12-22 1995-11-28 Imarx Pharmaceutical Corp. Methods of preparing gas-filled liposomes
US5580575A (en) 1989-12-22 1996-12-03 Imarx Pharmaceutical Corp. Therapeutic drug delivery systems
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
JP3220180B2 (ja) 1991-05-23 2001-10-22 三菱化学株式会社 薬剤含有タンパク質結合リポソーム
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
WO1993001286A2 (fr) 1991-06-28 1993-01-21 Massachusetts Institute Of Technology Therapie localisee utilisant des oligonucleotides
US5521291A (en) 1991-09-30 1996-05-28 Boehringer Ingelheim International, Gmbh Conjugates for introducing nucleic acid into higher eucaryotic cells
NZ244306A (en) 1991-09-30 1995-07-26 Boehringer Ingelheim Int Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation
FR2692265B1 (fr) 1992-05-25 1996-11-08 Centre Nat Rech Scient Composes biologiquement actifs de type phosphotriesters.
US6005079A (en) 1992-08-21 1999-12-21 Vrije Universiteit Brussels Immunoglobulins devoid of light chains
EP1498427B1 (fr) 1992-08-21 2009-12-16 Vrije Universiteit Brussel Immunoglobulines dépourvus de chaînes légères
US5583020A (en) 1992-11-24 1996-12-10 Ribozyme Pharmaceuticals, Inc. Permeability enhancers for negatively charged polynucleotides
JP3351476B2 (ja) 1993-01-22 2002-11-25 三菱化学株式会社 リン脂質誘導体及びそれを含有するリポソーム
US5395619A (en) 1993-03-03 1995-03-07 Liposome Technology, Inc. Lipid-polymer conjugates and liposomes
US5994618A (en) 1997-02-05 1999-11-30 Johns Hopkins University School Of Medicine Growth differentiation factor-8 transgenic mice
ES2201076T3 (es) 1993-03-19 2004-03-16 The Johns Hopkins University School Of Medicine Factor-8 de diferenciacion del crecimiento.
US5462854A (en) 1993-04-19 1995-10-31 Beckman Instruments, Inc. Inverse linkage oligonucleotides for chemical and enzymatic processes
DK0698097T3 (da) 1993-04-29 2001-10-08 Unilever Nv Produktion af antistoffer eller (funktionaliserede) fragmenter deraf afledt af Camelidae-immunoglobuliner med tung kæde
FR2705099B1 (fr) 1993-05-12 1995-08-04 Centre Nat Rech Scient Oligonucléotides phosphorothioates triesters et procédé de préparation.
US5534259A (en) 1993-07-08 1996-07-09 Liposome Technology, Inc. Polymer compound and coated particle composition
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5417978A (en) 1993-07-29 1995-05-23 Board Of Regents, The University Of Texas System Liposomal antisense methyl phosphonate oligonucleotides and methods for their preparation and use
US5595756A (en) 1993-12-22 1997-01-21 Inex Pharmaceuticals Corporation Liposomal compositions for enhanced retention of bioactive agents
US5543152A (en) 1994-06-20 1996-08-06 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5591721A (en) 1994-10-25 1997-01-07 Hybridon, Inc. Method of down-regulating gene expression
US5512295A (en) 1994-11-10 1996-04-30 The Board Of Trustees Of The Leland Stanford Junior University Synthetic liposomes for enhanced uptake and delivery
US5707618A (en) 1995-03-24 1998-01-13 Genzyme Corporation Adenovirus vectors for gene therapy
DK2111876T3 (da) 1995-12-18 2011-12-12 Angiodevice Internat Gmbh Tværbundne polymerpræparater og fremgangsmåder til anvendelse deraf
US6245747B1 (en) 1996-03-12 2001-06-12 The Board Of Regents Of The University Of Nebraska Targeted site specific antisense oligodeoxynucleotide delivery method
WO1999002667A1 (fr) * 1997-07-14 1999-01-21 University Of Liege Mutation du gene de la myostatine a l'origine de l'hypertrophie musculaire chez les mammiferes
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
AU776362B2 (en) 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
US6284882B1 (en) 1999-06-10 2001-09-04 Abbott Laboratories Myostatin gene promoter and inhibition of activation thereof
US6287860B1 (en) 2000-01-20 2001-09-11 Isis Pharmaceuticals, Inc. Antisense inhibition of MEKK2 expression
EP1191097A1 (fr) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction du "exon-skipping" dans des cellules eukaryotes
US20050124566A1 (en) 2001-05-18 2005-06-09 Sirna Therapeutics, Inc. RNA interference mediated inhibition of myostatin gene expression using short interfering nucleic acid (siNA)
CA2459347C (fr) 2001-09-04 2012-10-09 Exiqon A/S Compositions d'acides nucleiques bloques et leurs utilisations
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
KR100464261B1 (ko) 2002-01-24 2005-01-03 주식회사 파나진 Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
KR20030084444A (ko) 2002-04-26 2003-11-01 주식회사 파나진 Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
WO2004044140A2 (fr) 2002-11-05 2004-05-27 Isis Pharmaceticals, Inc. Composes oligomeres 2'-substitues et compositions destinees a etre utilisees dans des modulations genetiques
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
ES2351976T3 (es) * 2003-04-29 2011-02-14 Avi Biopharma, Inc. Composiciones para mejorar el transporte y la eficacia antisentido de análogos de ácidos nucleicos en células.
US7211668B2 (en) 2003-07-28 2007-05-01 Panagene, Inc. PNA monomer and precursor
SI2206781T1 (sl) 2004-06-28 2016-05-31 The University Of Western Australia Protismiselni oligonukleotidi za induciranje preskakovanja eksona in postopki njihove uporabe
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
NZ538097A (en) 2005-02-07 2006-07-28 Ovita Ltd Method and compositions for improving wound healing
ES2852549T3 (es) * 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
CA2605512A1 (fr) 2005-04-22 2006-10-26 Academisch Ziekenhuis Leiden Modulation de la reconnaissance d'exon dans un pre-arnm par interference avec la liaison de proteines sr et interference avec une structure d'arn secondaire
BRPI0610248A2 (pt) 2005-04-25 2010-06-08 Pfizer anticorpos contra miostatina, composição farmacêutica compreendendo os mesmos, linhas celulares que os produzem , moléculas de ácido nucleico codificantes dos mesmos bem como seus usos
CA2538208A1 (fr) 2005-05-04 2006-11-04 Universite Laval Modulation de la myostatine et utilisation de celle-ci dans le traitement de maladies musculaires axe sur la greffe de cellules
DE202005008426U1 (de) 2005-05-31 2005-09-15 Ricon Sieb Und Foerdertechnik Fördervorrichtung mit Fördertuch
US8361977B2 (en) 2005-06-23 2013-01-29 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of SMN2 splicing
WO2007003216A1 (fr) 2005-07-06 2007-01-11 Universidad Autónoma de Madrid Anticorps anti-récepteur ccr7 pour le traitement du cancer
ES2657400T3 (es) 2006-05-10 2018-03-05 Sarepta Therapeutics, Inc. Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos
EP1857548A1 (fr) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Procédé et moyen permettant d'induire un saut d'exon
US8097596B2 (en) * 2006-06-30 2012-01-17 Lakewood-Amedex, Inc. Compositions and methods for the treatment of muscle wasting
WO2008088895A2 (fr) 2007-01-18 2008-07-24 University Of Missouri-Columbia Mini/microgènes synthétiques de la dystrophine pour redonner de la no-synthase neuronale au sarcolemme
TW201940502A (zh) 2007-02-02 2019-10-16 美商艾瑟勒朗法瑪公司 衍生自ＡｃｔＲＩＩＢ的變體與其用途
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
WO2009008725A2 (fr) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molécules pour cibler des composés vers divers organes, tissus ou cellules tumorales sélectionnés
US7935816B2 (en) 2007-10-25 2011-05-03 Gene Tools, Llc Molecular transporter compositions comprising dendrimeric oligoguanidine with a triazine core that facilitate delivery into cells in vivo
DK2203173T3 (en) 2007-10-26 2016-02-29 Academisch Ziekenhuis Leiden Materials and methods for prevention of muscle diseases
US8076476B2 (en) 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
CA2884340C (fr) 2007-11-15 2017-07-25 Sarepta Therapeutics, Inc. Procede de synthese d'oligomeres morpholino
CA2711302C (fr) 2008-01-04 2016-11-08 Cue Acoustics, Inc. Dispositif audio dote d'une alimentation a commutation integree
EP2119783A1 (fr) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Procédé pour l'omission efficace de l'exon (44) dans la dystrophie musculaire de Duchenne et moyens connexes
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
LT3750552T (lt) 2008-08-14 2023-06-26 Acceleron Pharma Inc. Gdf gaudyklės
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP2762567B1 (fr) 2008-10-24 2016-07-13 Sarepta Therapeutics, Inc. Compositions de saut d'exon multiples pour dmd
CN102625840A (zh) 2009-04-10 2012-08-01 肌肉学研究协会 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法
WO2010120820A1 (fr) 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions et procédés pour moduler l'épissage de smn2
ES2593836T3 (es) 2009-04-24 2016-12-13 Biomarin Technologies B.V. Oligonucleótido que comprende una inosina para tratar la DMD
EA027071B1 (ru) 2009-04-27 2017-06-30 Новартис Аг АНТИТЕЛО К ActRIIB И СОДЕРЖАЩАЯ ЕГО КОМПОЗИЦИЯ
WO2010129406A2 (fr) * 2009-05-04 2010-11-11 The Johns Hopkins University Procédés de promotion de la croissance musculaire
PT3449926T (pt) 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito
SMT201800579T1 (it) 2009-11-12 2019-01-11 Univ Western Australia Molecole antisenso e metodi per trattare patologie
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
JP2013530154A (ja) 2010-05-28 2013-07-25 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
PL2623507T3 (pl) 2010-09-30 2017-03-31 Nippon Shinyaku Co., Ltd. Pochodna kwasu morfolinonukleinowego
EP2672977A1 (fr) 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Oligonucléotides antisens
WO2012150960A1 (fr) 2011-05-05 2012-11-08 Avi Biopharma, Inc. Conjugués peptides/oligonucléotides
US20130085139A1 (en) * 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
ES2535654T3 (es) 2011-10-13 2015-05-13 Association Institut De Myologie ADN triciclo-fosforotioato
BR112014011875B1 (pt) 2011-11-18 2022-01-04 Sarepta Therapeutics, Inc Oligonucleotídeos funcionalmente modificados e subunidades dos mesmos
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
CN108611349A (zh) 2011-12-28 2018-10-02 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
AU2013285698A1 (en) * 2012-07-03 2015-02-19 Biomarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
KR20150099804A (ko) 2012-12-20 2015-09-01 사렙타 쎄러퓨틱스 인코퍼레이티드 근위축증을 치료하기 위한 개선된 엑손 스키핑 조성물
AU2014236140B2 (en) 2013-03-14 2019-10-03 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
NZ732507A (en) * 2013-03-15 2018-08-31 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
WO2014172448A2 (fr) 2013-04-17 2014-10-23 Anaptysbio, Inc. Anticorps diriges contre le recepteur d'activine de type ii (actrii)
IL282239B2 (en) * 2013-09-05 2023-10-01 Sarepta Therapeutics Inc Antisense-induced exon2 inclusion in acid alpha-glucosidase
US10867398B2 (en) 2017-11-21 2020-12-15 Reliance Core Consulting LLC Methods, systems, apparatuses and devices for facilitating motion analysis in an environment

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US20190177723A1 (en) 2019-06-13
EP3858993A1 (fr) 2021-08-04
WO2017062835A3 (fr) 2017-06-08
WO2017062835A2 (fr) 2017-04-13
EP3359668A2 (fr) 2018-08-15
EP3359668A4 (fr) 2019-06-05
IL258069A (en) 2018-05-31
CN108699555A (zh) 2018-10-23
EA201890908A1 (ru) 2018-10-31

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