IL296255A - Gene therapy - Google Patents
Gene therapyInfo
- Publication number
- IL296255A IL296255A IL296255A IL29625522A IL296255A IL 296255 A IL296255 A IL 296255A IL 296255 A IL296255 A IL 296255A IL 29625522 A IL29625522 A IL 29625522A IL 296255 A IL296255 A IL 296255A
- Authority
- IL
- Israel
- Prior art keywords
- viral vector
- seq
- promoter
- aav
- col4a5
- Prior art date
Links
Classifications
-
- 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
-
- 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
- C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
- C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
-
- 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
- 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
- C12N2750/14145—Special targeting system for 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
- 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
- C12N2830/50—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)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biophysics (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Toxicology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB2003618.2A GB202003618D0 (en) | 2020-03-12 | 2020-03-12 | Gene Therapy |
| PCT/GB2021/050633 WO2021181118A1 (en) | 2020-03-12 | 2021-03-12 | Gene therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL296255A true IL296255A (en) | 2022-11-01 |
Family
ID=70453740
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL296255A IL296255A (en) | 2020-03-12 | 2021-03-12 | Gene therapy |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US12589167B2 (https=) |
| EP (1) | EP4118216A1 (https=) |
| JP (1) | JP2023517934A (https=) |
| CN (1) | CN115427578B (https=) |
| AU (1) | AU2021234169A1 (https=) |
| BR (1) | BR112022018036A2 (https=) |
| CA (1) | CA3171232A1 (https=) |
| GB (1) | GB202003618D0 (https=) |
| IL (1) | IL296255A (https=) |
| MX (1) | MX2022011295A (https=) |
| WO (1) | WO2021181118A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB202003618D0 (en) | 2020-03-12 | 2020-04-29 | Univ Bristol | Gene Therapy |
| EP4273243A1 (en) | 2022-05-02 | 2023-11-08 | Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca | Nucleic acid constructs and vectors for podocyte specific expression |
| GB202304841D0 (en) * | 2023-03-31 | 2023-05-17 | Purespring Therapeutics Ltd | Promoter |
| CN116590345B (zh) * | 2023-05-06 | 2024-01-30 | 北京中医药大学 | 永生化小鼠足细胞系及其制备方法、分化方法和应用 |
| GB202309913D0 (en) * | 2023-06-29 | 2023-08-16 | Purespring Therapeutics Ltd | Polynucleotides, avv vectors, and pharmaceutical compositions for treating nephrotic syndrome |
| GB202309908D0 (en) * | 2023-06-29 | 2023-08-16 | Purespring Therapeutics Ltd | Polynucleotides, aav vectors, and pharmaceutical compositions for treating complement-mediated kidney diseases |
| GB202309914D0 (en) * | 2023-06-29 | 2023-08-16 | Purespring Therapeutics Ltd | Promoters |
| GB202309909D0 (en) * | 2023-06-29 | 2023-08-16 | Purespring Therapeutics Ltd | Regulatory elements |
| WO2025168940A1 (en) * | 2024-02-07 | 2025-08-14 | Purespring Therapeutics Limited | Vectors |
| WO2025193938A1 (en) * | 2024-03-13 | 2025-09-18 | Oregon Health & Science University | Delivery of type iv collagen alpha chain using a split intein dual aav vector |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL332875A1 (en) | 1996-10-17 | 1999-10-25 | Oxford Biomedica Ltd | Retroviral vectors |
| US20060281090A1 (en) | 2003-05-01 | 2006-12-14 | University Of Washington Uw Tech Transfer- Invention Licensing | Capsid-modified adenovirus vectors and methods of using the same |
| WO2005012351A2 (en) | 2003-08-01 | 2005-02-10 | The Brigham And Women's Hospital, Inc. | Novel functions for decay accelerating factor (daf) in inflammation |
| EP2357254A1 (en) | 2005-02-14 | 2011-08-17 | University of Iowa Research Foundation | Methods and reagents for treatment and diagnosis of age-related macular degeneration |
| WO2007056435A2 (en) | 2005-11-08 | 2007-05-18 | The General Hospital Corporation | Dynamin mediated diseases and associated methods and products |
| WO2008136547A1 (en) | 2007-05-07 | 2008-11-13 | University Of Ulsan Foundation For Industry Cooperation | Method of preventing or treating body weight disorders by employing clusterin |
| ES2457822T3 (es) | 2007-11-08 | 2014-04-29 | The General Hospital Corporation | Procedimientos y composiciones de tratamiento de enfermedades proteinúricas |
| EP2601288B1 (en) | 2010-08-05 | 2016-04-06 | Wisconsin Alumni Research Foundation | Simplified basic media for human pluripotent cell culture |
| US8852936B2 (en) | 2012-09-21 | 2014-10-07 | Laurantis Pharma Oy | Autologous lymph node transfer in combination with VEGF-C or VEGF-D growth factor therapy to treat lymphedema and to improve reconstructive surgery |
| TW201506036A (zh) | 2013-08-14 | 2015-02-16 | Laurantis Pharma Oy | Vegf-c及ccbe1之治療用途 |
| CA2955481A1 (en) | 2014-07-25 | 2016-01-28 | Goldfinch Biopharma, Inc. | Collagen iv replacement |
| EP3526333A4 (en) * | 2016-10-13 | 2020-07-29 | University of Massachusetts | AAV CAPSIDE DESIGNS |
| CN107011424B (zh) | 2017-03-14 | 2020-07-03 | 华南师范大学 | 斜带石斑鱼补体c1inh基因、载体、重组菌株和蛋白及其应用 |
| GB201706808D0 (en) | 2017-04-28 | 2017-06-14 | Univ Newcastle | Modified complement proteins and uses thereof |
| EP3635121A1 (en) * | 2017-06-02 | 2020-04-15 | Institut National de la Sante et de la Recherche Medicale (INSERM) | Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders |
| CN111788311A (zh) | 2017-10-20 | 2020-10-16 | 双子治疗公司 | 用于治疗年龄相关性黄斑变性的组合物和方法 |
| RU2675861C1 (ru) | 2017-11-22 | 2018-12-25 | Лев Иосифович Шагам | Способ прогнозирования наличия мутации гена col4a5 у пациентов с предполагаемым диагнозом "синдром альпорта" |
| EP3856763A1 (en) * | 2018-09-28 | 2021-08-04 | Massachusetts Institute of Technology | Collagen-localized immunomodulatory molecules and methods thereof |
| EP3861010A1 (en) * | 2018-10-02 | 2021-08-11 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| CA3117551A1 (en) | 2018-10-23 | 2020-04-30 | Gemini Therapeutics Inc. | Compositions and methods for treating age-related macular degeneration and other diseases |
| RU2714763C1 (ru) | 2018-11-08 | 2020-02-19 | Генетик Диагностикс Энд Терапи 21 Лтд | Генотерапевтический ДНК-вектор для таргетной генной терапии, способ его получения (варианты), штамм для его производства, способ его получения |
| GB201900702D0 (en) | 2019-01-18 | 2019-03-06 | Univ Bristol | Therapy |
| GB202003618D0 (en) | 2020-03-12 | 2020-04-29 | Univ Bristol | Gene Therapy |
| GB202010009D0 (en) | 2020-06-30 | 2020-08-12 | Syncona Investment Man Ltd | Vector |
| GB202103470D0 (en) | 2021-03-12 | 2021-04-28 | Univ Bristol | Promoter |
-
2020
- 2020-03-12 GB GBGB2003618.2A patent/GB202003618D0/en not_active Ceased
-
2021
- 2021-03-12 MX MX2022011295A patent/MX2022011295A/es unknown
- 2021-03-12 AU AU2021234169A patent/AU2021234169A1/en active Pending
- 2021-03-12 IL IL296255A patent/IL296255A/en unknown
- 2021-03-12 BR BR112022018036A patent/BR112022018036A2/pt unknown
- 2021-03-12 CN CN202180020240.6A patent/CN115427578B/zh active Active
- 2021-03-12 CA CA3171232A patent/CA3171232A1/en active Pending
- 2021-03-12 EP EP21711944.5A patent/EP4118216A1/en active Pending
- 2021-03-12 US US17/909,839 patent/US12589167B2/en active Active
- 2021-03-12 JP JP2022554473A patent/JP2023517934A/ja active Pending
- 2021-03-12 WO PCT/GB2021/050633 patent/WO2021181118A1/en not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| EP4118216A1 (en) | 2023-01-18 |
| CA3171232A1 (en) | 2021-09-16 |
| US12589167B2 (en) | 2026-03-31 |
| CN115427578A (zh) | 2022-12-02 |
| WO2021181118A1 (en) | 2021-09-16 |
| AU2021234169A1 (en) | 2022-10-06 |
| US20240197917A1 (en) | 2024-06-20 |
| JP2023517934A (ja) | 2023-04-27 |
| MX2022011295A (es) | 2022-11-14 |
| BR112022018036A2 (pt) | 2022-11-01 |
| CN115427578B (zh) | 2026-03-17 |
| GB202003618D0 (en) | 2020-04-29 |
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