IL258069A - Preparations and methods for the treatment of Duchenne muscular dystrophy and related disorders - Google Patents
Preparations and methods for the treatment of Duchenne muscular dystrophy and related disordersInfo
- Publication number
- IL258069A IL258069A IL258069A IL25806918A IL258069A IL 258069 A IL258069 A IL 258069A IL 258069 A IL258069 A IL 258069A IL 25806918 A IL25806918 A IL 25806918A IL 258069 A IL258069 A IL 258069A
- Authority
- IL
- Israel
- Prior art keywords
- compositions
- methods
- related disorders
- muscular dystrophy
- duchenne muscular
- Prior art date
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
-
- 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)
- 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)
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 |
|---|---|
| IL258069A true IL258069A (en) | 2018-05-31 |
Family
ID=58488705
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL258069A IL258069A (en) | 2015-10-09 | 2018-03-13 | Preparations and methods for the treatment of Duchenne muscular dystrophy and related disorders |
| IL290160A IL290160A (en) | 2015-10-09 | 2022-01-27 | Preparations and methods for the treatment of Duchenne muscular dystrophy and related disorders |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL290160A IL290160A (en) | 2015-10-09 | 2022-01-27 | Preparations and methods for the treatment of Duchenne muscular dystrophy and related disorders |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20190177723A1 (cg-RX-API-DMAC7.html) |
| EP (2) | EP3858993A1 (cg-RX-API-DMAC7.html) |
| JP (2) | JP2018530560A (cg-RX-API-DMAC7.html) |
| CN (1) | CN108699555A (cg-RX-API-DMAC7.html) |
| BR (1) | BR112018007066A2 (cg-RX-API-DMAC7.html) |
| EA (1) | EA201890908A1 (cg-RX-API-DMAC7.html) |
| IL (2) | IL258069A (cg-RX-API-DMAC7.html) |
| MA (1) | MA45819A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017062835A2 (cg-RX-API-DMAC7.html) |
Families Citing this family (52)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2596506C (en) | 2005-02-09 | 2021-04-06 | Avi Biopharma, 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 |
| EP3478697A1 (en) * | 2016-06-30 | 2019-05-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| FI4122497T3 (fi) * | 2016-12-19 | 2024-06-04 | Sarepta Therapeutics Inc | Eksonin ohittavia oligomeerikonjugaatteja lihasdystrofiaan |
| LT3554553T (lt) * | 2016-12-19 | 2022-08-25 | Sarepta Therapeutics, Inc. | Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos |
| JP7160807B2 (ja) * | 2016-12-19 | 2022-10-25 | 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コンジュゲート |
| WO2019046755A1 (en) * | 2017-08-31 | 2019-03-07 | 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 |
| US20200362339A1 (en) * | 2017-09-25 | 2020-11-19 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers via fast-flow synthesis |
| 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 |
| JP2021511803A (ja) * | 2018-01-31 | 2021-05-13 | ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム | ヒト心筋細胞におけるジストロフィン変異を修正するための組成物および方法 |
| US20210102205A1 (en) * | 2018-04-26 | 2021-04-08 | 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 |
| US10758629B2 (en) * | 2018-05-29 | 2020-09-01 | 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 |
| US20210220386A1 (en) * | 2018-06-14 | 2021-07-22 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
| 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 |
| EA202190416A1 (ru) | 2018-08-02 | 2021-06-23 | Дайн Терапьютикс, Инк. | Мышечно-специфические комлексы и их применение для лечения плече-лопаточно-лицевой мышечной дистрофии |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| MX2021001281A (es) | 2018-08-02 | 2021-07-15 | Dyne Therapeutics Inc | Complejos dirigidos a músculos y sus usos para el tratamiento de distrofinopatías. |
| 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 |
| 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 |
| BR112021008069A2 (pt) * | 2018-11-02 | 2021-11-03 | Biomarin Tech Bv | Oligonucleotídeos antisense biespecíficos para exon skipping da distrofina |
| CN113383078A (zh) * | 2018-12-06 | 2021-09-10 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| KR20210109551A (ko) | 2018-12-07 | 2021-09-06 | 옥스포드 유니버시티 이노베이션 리미티드 | 링커 |
| WO2020123574A1 (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 |
| CA3137715A1 (en) | 2019-04-25 | 2020-10-29 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| 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 |
| EP4007633A4 (en) * | 2019-08-02 | 2024-05-08 | Research Institute at Nationwide Children's Hospital | EXON 44-TARGETED NUCLEIC ACIDS AND RECOMBINANT ADENOA-ASSOCIATED VIRUS FOR THE TREATMENT OF DYSTROPHIN-BASED MYOPATHIES |
| CN115175991A (zh) | 2019-10-16 | 2022-10-11 | 博德研究所 | 工程化肌肉靶向组合物 |
| AU2020392220A1 (en) * | 2019-11-27 | 2022-06-02 | Novartis Ag | Compounds and methods for the treatment of Duchenne muscular dystrophy |
| CN118109469A (zh) * | 2019-12-26 | 2024-05-31 | 日本新药株式会社 | 诱导外显子50的跳读的反义核酸 |
| MX2023003609A (es) * | 2020-09-30 | 2023-04-05 | Biomarin Tech Bv | Oligonucleotidos antisentido que se dirigen al exon 51 del gen de la distrofina. |
| CA3211057A1 (en) * | 2021-02-26 | 2022-09-01 | Knc Laboratories Co., Ltd. | Nucleic acid medicine expressing splicing variant of myostatin |
| EP4304628A2 (en) * | 2021-03-12 | 2024-01-17 | Pepgen Inc. | Methods of treating duchenne muscular dystrophy using peptide-oligonucleotide conjugates |
| 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 |
| 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 |
| KR20240155352A (ko) * | 2022-03-17 | 2024-10-28 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머 접합체 |
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| US20250288641A1 (en) * | 2022-04-27 | 2025-09-18 | The Curators Of The University Of Missouri | Micro-dystrophin for heart protection |
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- 2016-10-07 MA MA045819A patent/MA45819A/fr unknown
- 2016-10-07 WO PCT/US2016/056093 patent/WO2017062835A2/en not_active Ceased
- 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 EP EP21151113.4A patent/EP3858993A1/en not_active Withdrawn
- 2016-10-07 CN CN201680072146.4A patent/CN108699555A/zh active Pending
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2021
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| EP3359668A2 (en) | 2018-08-15 |
| EP3359668A4 (en) | 2019-06-05 |
| BR112018007066A2 (pt) | 2018-10-23 |
| MA45819A (fr) | 2018-08-15 |
| CN108699555A (zh) | 2018-10-23 |
| JP2018530560A (ja) | 2018-10-18 |
| EP3858993A1 (en) | 2021-08-04 |
| IL290160A (en) | 2022-03-01 |
| WO2017062835A3 (en) | 2017-06-08 |
| US20190177723A1 (en) | 2019-06-13 |
| WO2017062835A2 (en) | 2017-04-13 |
| JP2022017594A (ja) | 2022-01-25 |
| EA201890908A1 (ru) | 2018-10-31 |
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