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 1
- 239000000203 mixture Substances 0.000 title 1
- 201000006938 muscular dystrophy Diseases 0.000 title 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
-
- 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
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)
- Neurology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Cell Biology (AREA)
- Psychology (AREA)
- Proteomics, Peptides & Aminoacids (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)
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 (enExample) |
| EP (2) | EP3359668A4 (enExample) |
| JP (2) | JP2018530560A (enExample) |
| CN (1) | CN108699555A (enExample) |
| BR (1) | BR112018007066A2 (enExample) |
| EA (1) | EA201890908A1 (enExample) |
| IL (2) | IL258069A (enExample) |
| MA (1) | MA45819A (enExample) |
| WO (1) | WO2017062835A2 (enExample) |
Families Citing this family (53)
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| AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| 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 |
| TW201811807A (zh) * | 2016-06-30 | 2018-04-01 | 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 |
| US20180028554A1 (en) * | 2016-07-05 | 2018-02-01 | Biomarin Technologies B.V. | Oligomers Having Bicyclic Scaffold Moeities |
| MY195801A (en) * | 2016-12-19 | 2023-02-22 | Sarepta Therapeutics Inc | Exon Skipping Oligomer Conjugates for Muscular Dystrophy |
| RS63705B1 (sr) | 2016-12-19 | 2022-11-30 | Sarepta Therapeutics Inc | Oligomerni konjugati za preskakanje egzona za mišićnu distrofiju |
| PT3554552T (pt) * | 2016-12-19 | 2022-10-03 | Sarepta Therapeutics Inc | Conjugados de oligómero de skipping de exão para distrofia muscular |
| EP3675836A4 (en) * | 2017-08-31 | 2021-05-26 | Sarepta Therapeutics, Inc. | METHODS FOR TREATING MUSCLE DYSTROPHY |
| 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 |
| EP3717008A1 (en) | 2017-12-01 | 2020-10-07 | Merus N.V. | Use of bispecific antibody and il-15 for combination therapy |
| EA202091828A1 (ru) * | 2018-01-31 | 2021-05-24 | Дзе Борд Оф Риджентс Оф Дзе Юниверсити Оф Техас Систем | Композиции и способы коррекции мутаций дистрофина в кардиомиоцитах человека |
| WO2019209764A2 (en) * | 2018-04-26 | 2019-10-31 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
| US12391942B2 (en) | 2018-05-11 | 2025-08-19 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| US10765760B2 (en) * | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| JP2021526796A (ja) * | 2018-06-13 | 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 |
| EP4219717A3 (en) * | 2018-06-13 | 2023-12-20 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| JP7579147B2 (ja) * | 2018-06-14 | 2024-11-07 | 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 |
| 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 |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
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| JP7611825B2 (ja) | 2018-08-02 | 2025-01-10 | 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それらの使用 |
| GB201812980D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| GB201812972D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| EP3874044A1 (en) * | 2018-11-02 | 2021-09-08 | BioMarin Technologies B.V. | Bispecific antisense oligonucleotides for dystrophin exon skipping |
| US20220186217A1 (en) * | 2018-12-06 | 2022-06-16 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| BR112021010982A2 (pt) | 2018-12-07 | 2021-08-31 | Oxford University Innovation Limited | Ligantes |
| CA3122281A1 (en) * | 2018-12-13 | 2020-06-18 | 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 |
| JP7687682B2 (ja) * | 2019-04-08 | 2025-06-03 | 国立大学法人東京科学大学 | 筋疾患治療用医薬組成物 |
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| KR20220061915A (ko) * | 2019-05-06 | 2022-05-13 | 안티센스 테라퓨틱스 엘티디 | Cd49d에 대한 억제성 올리고뉴클레오티드를 사용하는 근이영양증의 치료 방법 |
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| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| AU2020392220A1 (en) * | 2019-11-27 | 2022-06-02 | Novartis Ag | Compounds and methods for the treatment of Duchenne muscular dystrophy |
| JPWO2021132591A1 (enExample) * | 2019-12-26 | 2021-07-01 | ||
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| WO2022192749A2 (en) * | 2021-03-12 | 2022-09-15 | Pepgen Inc. | Methods of treating duchenne muscular dystrophy using peptide-oligonucleotide conjugates |
| AU2022309028A1 (en) | 2021-07-09 | 2024-01-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| 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 |
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-
2016
- 2016-10-07 US US15/765,466 patent/US20190177723A1/en not_active Abandoned
- 2016-10-07 EA EA201890908A patent/EA201890908A1/ru unknown
- 2016-10-07 BR BR112018007066A patent/BR112018007066A2/pt not_active Application Discontinuation
- 2016-10-07 EP EP16854468.2A patent/EP3359668A4/en not_active Withdrawn
- 2016-10-07 MA MA045819A patent/MA45819A/fr unknown
- 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 JP JP2018517603A patent/JP2018530560A/ja not_active Withdrawn
- 2016-10-07 WO PCT/US2016/056093 patent/WO2017062835A2/en not_active Ceased
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2018
- 2018-03-13 IL IL258069A patent/IL258069A/en unknown
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2021
- 2021-11-15 JP JP2021185544A patent/JP2022017594A/ja active Pending
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2022
- 2022-01-27 IL IL290160A patent/IL290160A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| MA45819A (fr) | 2018-08-15 |
| EP3858993A1 (en) | 2021-08-04 |
| WO2017062835A3 (en) | 2017-06-08 |
| EP3359668A2 (en) | 2018-08-15 |
| BR112018007066A2 (pt) | 2018-10-23 |
| IL290160A (en) | 2022-03-01 |
| JP2022017594A (ja) | 2022-01-25 |
| EP3359668A4 (en) | 2019-06-05 |
| IL258069A (en) | 2018-05-31 |
| US20190177723A1 (en) | 2019-06-13 |
| CN108699555A (zh) | 2018-10-23 |
| JP2018530560A (ja) | 2018-10-18 |
| WO2017062835A2 (en) | 2017-04-13 |
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