EA201890908A1 - Композиции и способы для лечения мышечной дистрофии дюшенна и сходных нарушений - Google Patents
Композиции и способы для лечения мышечной дистрофии дюшенна и сходных нарушенийInfo
- Publication number
- EA201890908A1 EA201890908A1 EA201890908A EA201890908A EA201890908A1 EA 201890908 A1 EA201890908 A1 EA 201890908A1 EA 201890908 A EA201890908 A EA 201890908A EA 201890908 A EA201890908 A EA 201890908A EA 201890908 A1 EA201890908 A1 EA 201890908A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- treatment
- muscular dystrophy
- compositions
- methods
- young
- Prior art date
Links
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title abstract 2
- 201000006938 muscular dystrophy Diseases 0.000 title 1
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 abstract 2
- 208000035475 disorder Diseases 0.000 abstract 2
- 230000000692 anti-sense effect Effects 0.000 abstract 1
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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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
- C12N2320/33—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
Изобретение относится к композициям и способам для лечения мышечной дистрофии Дюшенна и сходных нарушений. Описаны модифицированные антисмысловые олигомеры для лечения мышечной дистрофии Дюшенна и сходных нарушений.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562239812P | 2015-10-09 | 2015-10-09 | |
PCT/US2016/056093 WO2017062835A2 (en) | 2015-10-09 | 2016-10-07 | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
Publications (1)
Publication Number | Publication Date |
---|---|
EA201890908A1 true EA201890908A1 (ru) | 2018-10-31 |
Family
ID=58488705
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA201890908A EA201890908A1 (ru) | 2015-10-09 | 2016-10-07 | Композиции и способы для лечения мышечной дистрофии дюшенна и сходных нарушений |
Country Status (9)
Country | Link |
---|---|
US (1) | US20190177723A1 (ru) |
EP (2) | EP3858993A1 (ru) |
JP (2) | JP2018530560A (ru) |
CN (1) | CN108699555A (ru) |
BR (1) | BR112018007066A2 (ru) |
EA (1) | EA201890908A1 (ru) |
IL (2) | IL258069A (ru) |
MA (1) | MA45819A (ru) |
WO (1) | WO2017062835A2 (ru) |
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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 |
KR20190024977A (ko) * | 2016-06-30 | 2019-03-08 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 |
KR20230110811A (ko) * | 2016-12-19 | 2023-07-25 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
PL3554554T3 (pl) * | 2016-12-19 | 2022-12-19 | Sarepta Therapeutics, Inc. | Koniugaty oligomerów z pominięciem egzonu w dystrofii mięśniowej |
CA3047010A1 (en) | 2016-12-19 | 2018-06-28 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
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EA201991450A1 (ru) * | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
ES2963336T3 (es) * | 2017-09-25 | 2024-03-26 | Sarepta Therapeutics Inc | Procesos para preparar oligómeros de fosforodiamidato morfolino mediante síntesis de flujo rápido |
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CN112218664A (zh) * | 2018-05-11 | 2021-01-12 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
US10765760B2 (en) * | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
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-
2016
- 2016-10-07 CN CN201680072146.4A patent/CN108699555A/zh active Pending
- 2016-10-07 EP EP21151113.4A patent/EP3858993A1/en not_active Withdrawn
- 2016-10-07 WO PCT/US2016/056093 patent/WO2017062835A2/en active Application Filing
- 2016-10-07 MA MA045819A patent/MA45819A/fr unknown
- 2016-10-07 EA EA201890908A patent/EA201890908A1/ru unknown
- 2016-10-07 US US15/765,466 patent/US20190177723A1/en not_active Abandoned
- 2016-10-07 JP JP2018517603A patent/JP2018530560A/ja not_active Withdrawn
- 2016-10-07 BR BR112018007066A patent/BR112018007066A2/pt not_active Application Discontinuation
- 2016-10-07 EP EP16854468.2A patent/EP3359668A4/en not_active Withdrawn
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2018
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2021
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IL258069A (en) | 2018-05-31 |
MA45819A (fr) | 2018-08-15 |
BR112018007066A2 (pt) | 2018-10-23 |
WO2017062835A3 (en) | 2017-06-08 |
CN108699555A (zh) | 2018-10-23 |
JP2018530560A (ja) | 2018-10-18 |
US20190177723A1 (en) | 2019-06-13 |
EP3359668A2 (en) | 2018-08-15 |
IL290160A (en) | 2022-03-01 |
JP2022017594A (ja) | 2022-01-25 |
EP3359668A4 (en) | 2019-06-05 |
EP3858993A1 (en) | 2021-08-04 |
WO2017062835A2 (en) | 2017-04-13 |
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