BR112023004605A2 - Vírus adeno-associado recombinante (raav) codificando gjb2 e usos do mesmo - Google Patents
Vírus adeno-associado recombinante (raav) codificando gjb2 e usos do mesmoInfo
- Publication number
- BR112023004605A2 BR112023004605A2 BR112023004605A BR112023004605A BR112023004605A2 BR 112023004605 A2 BR112023004605 A2 BR 112023004605A2 BR 112023004605 A BR112023004605 A BR 112023004605A BR 112023004605 A BR112023004605 A BR 112023004605A BR 112023004605 A2 BR112023004605 A2 BR 112023004605A2
- Authority
- BR
- Brazil
- Prior art keywords
- gjb2
- encoding
- protein
- raav
- associated virus
- Prior art date
Links
- 102100037156 Gap junction beta-2 protein Human genes 0.000 title abstract 12
- 101000954092 Homo sapiens Gap junction beta-2 protein Proteins 0.000 title abstract 11
- 241000702421 Dependoparvovirus Species 0.000 title abstract 2
- 210000004027 cell Anatomy 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 101150028074 2 gene Proteins 0.000 abstract 1
- 102000010970 Connexin Human genes 0.000 abstract 1
- 108050001175 Connexin Proteins 0.000 abstract 1
- 108010069156 Connexin 26 Proteins 0.000 abstract 1
- 206010011878 Deafness Diseases 0.000 abstract 1
- 208000031514 autosomal recessive nonsyndromic hearing loss 1A Diseases 0.000 abstract 1
- 210000003027 ear inner Anatomy 0.000 abstract 1
- 239000003623 enhancer Substances 0.000 abstract 1
- 210000000630 fibrocyte Anatomy 0.000 abstract 1
- 231100000888 hearing loss Toxicity 0.000 abstract 1
- 230000010370 hearing loss Effects 0.000 abstract 1
- 208000016354 hearing loss disease Diseases 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 201000006790 nonsyndromic deafness Diseases 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 210000002985 organ of corti Anatomy 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
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
- C12N15/63—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
-
- 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
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- 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
-
- 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
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063078233P | 2020-09-14 | 2020-09-14 | |
| US202163161619P | 2021-03-16 | 2021-03-16 | |
| PCT/US2021/050205 WO2022056444A1 (en) | 2020-09-14 | 2021-09-14 | Recombinant adeno associated virus (raav) encoding gjb2 and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112023004605A2 true BR112023004605A2 (pt) | 2023-04-11 |
Family
ID=80631939
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112023004605A BR112023004605A2 (pt) | 2020-09-14 | 2021-09-14 | Vírus adeno-associado recombinante (raav) codificando gjb2 e usos do mesmo |
Country Status (10)
| Country | Link |
|---|---|
| EP (1) | EP4211151A4 (https=) |
| JP (1) | JP2023541443A (https=) |
| KR (1) | KR20230069157A (https=) |
| AU (1) | AU2021339843A1 (https=) |
| BR (1) | BR112023004605A2 (https=) |
| CA (1) | CA3191533A1 (https=) |
| IL (1) | IL301057A (https=) |
| MX (1) | MX2023002978A (https=) |
| TW (1) | TW202227476A (https=) |
| WO (1) | WO2022056444A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2021067448A1 (en) * | 2019-09-30 | 2021-04-08 | Applied Genetic Technologies Corporation | Adeno-associated virus (aav) systems for treatment of genetic hearing loss |
| WO2024011224A2 (en) * | 2022-07-08 | 2024-01-11 | The Trustees Of Columbia University In The City Of New York | Regulatory element for cell type specific expression of genes in spinal motor neurons |
| WO2024218192A1 (en) * | 2023-04-18 | 2024-10-24 | Uniqure Biopharma B.V. | Novel neurotropic adeno-associated virus capsids with detargeting of peripheral organs |
| EP4713463A1 (en) * | 2023-05-18 | 2026-03-25 | Decibel Therapeutics, Inc. | Compositions and methods for treating gjb2-related hearing loss |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1308458A1 (en) * | 2001-10-30 | 2003-05-07 | GenOdyssee | New polynucleotides and polypeptides of the CX26 gene |
| DE602005019174D1 (de) * | 2004-06-17 | 2010-03-18 | Epigenomics Ag | Zusammensetzungen und verfahren zur verhinderung des mitschleppens einer kontamination in nukleinsäure-amplifikationsreaktionen |
| CN101389769A (zh) * | 2005-12-28 | 2009-03-18 | 森托科尔公司 | 用于评价和治疗银屑病及相关疾病的标记与方法 |
| WO2019200016A1 (en) * | 2018-04-10 | 2019-10-17 | President And Fellows Of Harvard College | Aav vectors encoding clarin-1 or gjb2 and uses thereof |
| WO2020077295A1 (en) * | 2018-10-11 | 2020-04-16 | Decibel Therapeutics, Inc. | Aav1 vectors and uses thereof for treatment of otic indications |
| EP4063510A1 (en) * | 2018-11-07 | 2022-09-28 | Akouos, Inc. | Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear |
| WO2021067448A1 (en) * | 2019-09-30 | 2021-04-08 | Applied Genetic Technologies Corporation | Adeno-associated virus (aav) systems for treatment of genetic hearing loss |
| WO2021231808A2 (en) * | 2020-05-13 | 2021-11-18 | Akouos, Inc. | Compositions and methods for treating gjb2-associated hearing loss |
| MX2023013394A (es) * | 2021-05-13 | 2023-12-14 | Akouos Inc | Composiciones de suministro de terapia genica y metodos para tratar perdida auditiva. |
-
2021
- 2021-09-14 TW TW110134291A patent/TW202227476A/zh unknown
- 2021-09-14 JP JP2023516689A patent/JP2023541443A/ja active Pending
- 2021-09-14 IL IL301057A patent/IL301057A/en unknown
- 2021-09-14 EP EP21867807.6A patent/EP4211151A4/en active Pending
- 2021-09-14 MX MX2023002978A patent/MX2023002978A/es unknown
- 2021-09-14 BR BR112023004605A patent/BR112023004605A2/pt unknown
- 2021-09-14 WO PCT/US2021/050205 patent/WO2022056444A1/en not_active Ceased
- 2021-09-14 CA CA3191533A patent/CA3191533A1/en active Pending
- 2021-09-14 AU AU2021339843A patent/AU2021339843A1/en active Pending
- 2021-09-14 KR KR1020237012321A patent/KR20230069157A/ko active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CA3191533A1 (en) | 2022-03-17 |
| EP4211151A1 (en) | 2023-07-19 |
| MX2023002978A (es) | 2023-06-01 |
| KR20230069157A (ko) | 2023-05-18 |
| TW202227476A (zh) | 2022-07-16 |
| AU2021339843A1 (en) | 2023-04-06 |
| JP2023541443A (ja) | 2023-10-02 |
| EP4211151A4 (en) | 2025-03-26 |
| IL301057A (en) | 2023-05-01 |
| WO2022056444A1 (en) | 2022-03-17 |
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