MX2024008939A - Moduladores de oligonucleotidos activadores de la expresion de la utrofina. - Google Patents
Moduladores de oligonucleotidos activadores de la expresion de la utrofina.Info
- Publication number
- MX2024008939A MX2024008939A MX2024008939A MX2024008939A MX2024008939A MX 2024008939 A MX2024008939 A MX 2024008939A MX 2024008939 A MX2024008939 A MX 2024008939A MX 2024008939 A MX2024008939 A MX 2024008939A MX 2024008939 A MX2024008939 A MX 2024008939A
- Authority
- MX
- Mexico
- Prior art keywords
- nucleic acid
- strand
- oligonucleotide
- acid strand
- activating
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 4
- 108010075653 Utrophin Proteins 0.000 title abstract 2
- 102000011856 Utrophin Human genes 0.000 title 1
- 230000003213 activating effect Effects 0.000 title 1
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 abstract 4
- 108020004707 nucleic acids Proteins 0.000 abstract 4
- 150000007523 nucleic acids Chemical class 0.000 abstract 4
- 102000039446 nucleic acids Human genes 0.000 abstract 4
- 201000006935 Becker muscular dystrophy Diseases 0.000 abstract 2
- 230000000692 anti-sense effect Effects 0.000 abstract 2
- 239000002773 nucleotide Substances 0.000 abstract 2
- 125000003729 nucleotide group Chemical group 0.000 abstract 2
- 102000001039 Dystrophin Human genes 0.000 abstract 1
- 108010069091 Dystrophin Proteins 0.000 abstract 1
- 108700039691 Genetic Promoter Regions Proteins 0.000 abstract 1
- 230000002950 deficient Effects 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
- C07K14/4708—Duchenne dystrophy
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN2022072958 | 2022-01-20 | ||
| PCT/CN2023/072076 WO2023138502A1 (en) | 2022-01-20 | 2023-01-13 | Oligonucleotide modulators activating utrophin expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2024008939A true MX2024008939A (es) | 2024-07-29 |
Family
ID=87347803
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2024008939A MX2024008939A (es) | 2022-01-20 | 2023-01-13 | Moduladores de oligonucleotidos activadores de la expresion de la utrofina. |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20250101421A1 (https=) |
| EP (1) | EP4466359A1 (https=) |
| JP (1) | JP2025504474A (https=) |
| KR (1) | KR20250005567A (https=) |
| CN (1) | CN118829728A (https=) |
| AU (1) | AU2023209973A1 (https=) |
| CA (1) | CA3249545A1 (https=) |
| MX (1) | MX2024008939A (https=) |
| TW (1) | TW202338088A (https=) |
| WO (1) | WO2023138502A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025201529A1 (en) * | 2024-03-29 | 2025-10-02 | Ractigen Therapeutics | Compounds for synthesizing modified small activating rna molecules, modified small activating rna molecules and uses thereof |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP5931720B2 (ja) * | 2009-05-08 | 2016-06-08 | クルナ・インコーポレーテッド | Ｄｍｄファミリーに対する天然アンチセンス転写物の抑制によるジストロフィンファミリー関連疾患の治療 |
| EA201492122A1 (ru) * | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии utrn |
| US20170044540A1 (en) * | 2014-04-22 | 2017-02-16 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| WO2021230385A1 (en) * | 2020-05-15 | 2021-11-18 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting utrophin gene |
-
2023
- 2023-01-13 CN CN202380018216.8A patent/CN118829728A/zh active Pending
- 2023-01-13 MX MX2024008939A patent/MX2024008939A/es unknown
- 2023-01-13 US US18/730,577 patent/US20250101421A1/en active Pending
- 2023-01-13 CA CA3249545A patent/CA3249545A1/en active Pending
- 2023-01-13 WO PCT/CN2023/072076 patent/WO2023138502A1/en not_active Ceased
- 2023-01-13 EP EP23742817.2A patent/EP4466359A1/en active Pending
- 2023-01-13 JP JP2024543108A patent/JP2025504474A/ja active Pending
- 2023-01-13 AU AU2023209973A patent/AU2023209973A1/en active Pending
- 2023-01-13 KR KR1020247027787A patent/KR20250005567A/ko active Pending
- 2023-01-19 TW TW112102859A patent/TW202338088A/zh unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2023209973A1 (en) | 2024-09-05 |
| TW202338088A (zh) | 2023-10-01 |
| JP2025504474A (ja) | 2025-02-12 |
| KR20250005567A (ko) | 2025-01-09 |
| EP4466359A1 (en) | 2024-11-27 |
| WO2023138502A1 (en) | 2023-07-27 |
| US20250101421A1 (en) | 2025-03-27 |
| CN118829728A (zh) | 2024-10-22 |
| CA3249545A1 (en) | 2023-07-27 |
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