CO2024012057A2 - Elementos reguladores de gjb2 y usos de los mismos - Google Patents
Elementos reguladores de gjb2 y usos de los mismosInfo
- Publication number
- CO2024012057A2 CO2024012057A2 CONC2024/0012057A CO2024012057A CO2024012057A2 CO 2024012057 A2 CO2024012057 A2 CO 2024012057A2 CO 2024012057 A CO2024012057 A CO 2024012057A CO 2024012057 A2 CO2024012057 A2 CO 2024012057A2
- Authority
- CO
- Colombia
- Prior art keywords
- gjb2
- promoters
- hearing loss
- regulatory elements
- enhancers
- Prior art date
Links
- 102100037156 Gap junction beta-2 protein Human genes 0.000 title abstract 6
- 101000954092 Homo sapiens Gap junction beta-2 protein Proteins 0.000 title abstract 6
- 230000001105 regulatory effect Effects 0.000 title 1
- 206010011878 Deafness Diseases 0.000 abstract 2
- 210000004027 cell Anatomy 0.000 abstract 2
- 239000003623 enhancer Substances 0.000 abstract 2
- 230000010370 hearing loss Effects 0.000 abstract 2
- 231100000888 hearing loss Toxicity 0.000 abstract 2
- 208000016354 hearing loss disease Diseases 0.000 abstract 2
- 101150034593 Gjb2 gene Proteins 0.000 abstract 1
- 108700019146 Transgenes Proteins 0.000 abstract 1
- 102000040430 polynucleotide Human genes 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 239000002157 polynucleotide Substances 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
Classifications
-
- 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
-
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0046—Ear
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Immunology (AREA)
- Marine Sciences & Fisheries (AREA)
- Toxicology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263306928P | 2022-02-04 | 2022-02-04 | |
| US202263306941P | 2022-02-04 | 2022-02-04 | |
| PCT/US2023/061953 WO2023150689A2 (en) | 2022-02-04 | 2023-02-03 | Gjb2 regulatory elements and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2024012057A2 true CO2024012057A2 (es) | 2024-09-30 |
Family
ID=87552983
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2024/0012057A CO2024012057A2 (es) | 2022-02-04 | 2024-09-04 | Elementos reguladores de gjb2 y usos de los mismos |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20250127928A1 (https=) |
| EP (1) | EP4473005A4 (https=) |
| JP (1) | JP2025506419A (https=) |
| KR (1) | KR20240156606A (https=) |
| AU (1) | AU2023214481A1 (https=) |
| CA (1) | CA3250345A1 (https=) |
| CL (1) | CL2024002306A1 (https=) |
| CO (1) | CO2024012057A2 (https=) |
| IL (1) | IL314705A (https=) |
| MX (1) | MX2024009520A (https=) |
| WO (1) | WO2023150689A2 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2026025095A2 (en) | 2024-07-26 | 2026-01-29 | Regeneron Pharmaceuticals, Inc. | Otic drug delivery system and methods |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3159549A1 (en) * | 2019-10-30 | 2021-05-06 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| EP4164695A4 (en) * | 2020-05-13 | 2024-06-05 | Akouos, Inc. | COMPOSITIONS AND METHODS FOR TREATING GJB2-ASSOCIATED HEARING LOSS |
-
2023
- 2023-02-03 CA CA3250345A patent/CA3250345A1/en active Pending
- 2023-02-03 AU AU2023214481A patent/AU2023214481A1/en active Pending
- 2023-02-03 IL IL314705A patent/IL314705A/en unknown
- 2023-02-03 MX MX2024009520A patent/MX2024009520A/es unknown
- 2023-02-03 EP EP23750443.6A patent/EP4473005A4/en active Pending
- 2023-02-03 US US18/835,452 patent/US20250127928A1/en active Pending
- 2023-02-03 WO PCT/US2023/061953 patent/WO2023150689A2/en not_active Ceased
- 2023-02-03 KR KR1020247029441A patent/KR20240156606A/ko active Pending
- 2023-02-03 JP JP2024546193A patent/JP2025506419A/ja active Pending
-
2024
- 2024-07-31 CL CL2024002306A patent/CL2024002306A1/es unknown
- 2024-09-04 CO CONC2024/0012057A patent/CO2024012057A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CA3250345A1 (en) | 2023-08-10 |
| CL2024002306A1 (es) | 2024-12-27 |
| JP2025506419A (ja) | 2025-03-11 |
| IL314705A (en) | 2024-10-01 |
| MX2024009520A (es) | 2024-09-11 |
| WO2023150689A2 (en) | 2023-08-10 |
| WO2023150689A8 (en) | 2023-10-12 |
| KR20240156606A (ko) | 2024-10-30 |
| EP4473005A4 (en) | 2026-02-18 |
| WO2023150689A3 (en) | 2023-11-16 |
| US20250127928A1 (en) | 2025-04-24 |
| AU2023214481A1 (en) | 2024-08-08 |
| EP4473005A2 (en) | 2024-12-11 |
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