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 associésInfo
- 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
Links
- 238000000034 method Methods 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title abstract 2
- 210000003205 muscle Anatomy 0.000 title 1
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 208000035475 disorder Diseases 0.000 abstract 2
- 230000000692 anti-sense effect Effects 0.000 abstract 1
Classifications
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- 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
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- 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)
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
Landscapes
- 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.
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) | EP3858993A1 (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 (40)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA2596506C (fr) | 2005-02-09 | 2021-04-06 | Avi Biopharma, Inc. | Composition antisens et methode permettant de traiter une atrophie musculaire |
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 |
MX2018016052A (es) * | 2016-06-30 | 2019-05-02 | Sarepta Therapeutics Inc | Oligomeros de omision de exon para distrofia muscular. |
KR102552428B1 (ko) * | 2016-12-19 | 2023-07-06 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
MX2019006879A (es) | 2016-12-19 | 2019-08-01 | Sarepta Therapeutics Inc | Conjugados de oligomeros de omision de exon para distrofia muscular. |
KR102639633B1 (ko) * | 2016-12-19 | 2024-02-26 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
CA3073736A1 (fr) * | 2017-08-31 | 2019-03-07 | Sarepta Therapeutics, Inc. | Methodes permettant de traiter la dystrophie musculaire |
EA201991450A1 (ru) * | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
JP7218361B2 (ja) * | 2017-09-25 | 2023-02-06 | サレプタ セラピューティクス, インコーポレイテッド | 高速流動合成を介してホスホロジアミデートモルホリノオリゴマーを調製するための方法 |
CN111432838A (zh) | 2017-12-01 | 2020-07-17 | 美勒斯公司 | 使用双特异性抗体和il-15进行联合治疗 |
US20200370042A1 (en) * | 2018-01-31 | 2020-11-26 | The Board Of Regents Of The University Of Texas System | Compositions and methods for correcting dystrophin mutations in human cardiomyocytes |
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 |
AU2019265904A1 (en) * | 2018-05-11 | 2020-11-12 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
US10758629B2 (en) * | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
WO2019241385A2 (fr) * | 2018-06-13 | 2019-12-19 | Sarepta Therapeutics, Inc. | Oligomères induisant un saut d'exon pour la dystrophie musculaire |
EP3806868A4 (fr) * | 2018-06-13 | 2022-06-22 | Sarepta Therapeutics, Inc. | Oligomères induisant un saut d'exon pour la dystrophie musculaire |
JP2021526807A (ja) * | 2018-06-14 | 2021-10-11 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート |
TW202020153A (zh) * | 2018-07-27 | 2020-06-01 | 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 |
US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
AU2019316103A1 (en) | 2018-08-02 | 2021-03-11 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
AU2019312692A1 (en) | 2018-08-02 | 2021-03-11 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
CA3118167A1 (fr) * | 2018-11-02 | 2020-05-07 | Biomarin Technologies B.V. | Oligonucleotides antisens bispecifiques pour le saut d'exon de la dystrophine |
US20220186217A1 (en) * | 2018-12-06 | 2022-06-16 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
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 |
US20220175817A1 (en) * | 2019-04-08 | 2022-06-09 | National University Corporation Tokyo Medical And Dental University | Pharmaceutical Composition for Treating Muscle Disease |
JP2022532098A (ja) * | 2019-05-06 | 2022-07-13 | アンチセンス セラピューティクス リミテッド | CD49dに対する阻害オリゴヌクレオチドを使用して筋ジストロフィーを治療する方法 |
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 |
KR20220147575A (ko) * | 2019-11-27 | 2022-11-03 | 디티엑스 파마, 인크. | 뒤시엔느 근위축증의 치료를 위한 화합물 및 방법 |
WO2021132591A1 (fr) * | 2019-12-26 | 2021-07-01 | 日本新薬株式会社 | Acide nucléique antisens qui induit le saut de l'exon 50 |
CA3193919A1 (fr) * | 2020-09-30 | 2022-04-07 | Biomarin Technologies B.V. | Oligonucleotide anti-sens ciblant l'exon 51 du gene de la dystrophine |
JP2024511955A (ja) * | 2021-03-12 | 2024-03-18 | ペプゲン インコーポレイテッド. | ペプチド-オリゴヌクレオチド複合体を用いるデュシェンヌ型筋ジストロフィーの治療法 |
US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
WO2023283623A1 (fr) * | 2021-07-09 | 2023-01-12 | Dyne Therapeutics, Inc. | Complexes ciblant le muscle et formulations de traitement de dystrophinopathies |
US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
WO2023178230A1 (fr) * | 2022-03-17 | 2023-09-21 | Sarepta Therapeutics, Inc. | Conjugués d'oligomère morpholino phosphorodiamidate |
US11931421B2 (en) | 2022-04-15 | 2024-03-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
WO2023212193A1 (fr) * | 2022-04-27 | 2023-11-02 | The Curators Of The University Of Missouri | Micro-dystrophine pour protection cardiaque |
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-
2016
- 2016-10-07 CN CN201680072146.4A patent/CN108699555A/zh active Pending
- 2016-10-07 US US15/765,466 patent/US20190177723A1/en not_active Abandoned
- 2016-10-07 EP EP21151113.4A patent/EP3858993A1/fr not_active Withdrawn
- 2016-10-07 JP JP2018517603A patent/JP2018530560A/ja not_active Withdrawn
- 2016-10-07 EA EA201890908A patent/EA201890908A1/ru unknown
- 2016-10-07 WO PCT/US2016/056093 patent/WO2017062835A2/fr active Application Filing
- 2016-10-07 EP EP16854468.2A patent/EP3359668A4/fr not_active Withdrawn
- 2016-10-07 MA MA045819A patent/MA45819A/fr unknown
- 2016-10-07 BR BR112018007066A patent/BR112018007066A2/pt not_active Application Discontinuation
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2018
- 2018-03-13 IL IL258069A patent/IL258069A/en unknown
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2021
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2022
- 2022-01-27 IL IL290160A patent/IL290160A/en unknown
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Publication number | Publication date |
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BR112018007066A2 (pt) | 2018-10-23 |
US20190177723A1 (en) | 2019-06-13 |
EP3359668A2 (fr) | 2018-08-15 |
WO2017062835A3 (fr) | 2017-06-08 |
IL290160A (en) | 2022-03-01 |
JP2018530560A (ja) | 2018-10-18 |
WO2017062835A2 (fr) | 2017-04-13 |
IL258069A (en) | 2018-05-31 |
JP2022017594A (ja) | 2022-01-25 |
CN108699555A (zh) | 2018-10-23 |
EA201890908A1 (ru) | 2018-10-31 |
EP3359668A4 (fr) | 2019-06-05 |
EP3858993A1 (fr) | 2021-08-04 |
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