CA3171232A1 - Gene therapy - Google Patents
Gene therapy Download PDFInfo
- Publication number
- CA3171232A1 CA3171232A1 CA3171232A CA3171232A CA3171232A1 CA 3171232 A1 CA3171232 A1 CA 3171232A1 CA 3171232 A CA3171232 A CA 3171232A CA 3171232 A CA3171232 A CA 3171232A CA 3171232 A1 CA3171232 A1 CA 3171232A1
- Authority
- CA
- Canada
- Prior art keywords
- viral vector
- seq
- promoter
- aav
- col4a5
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB2003618.2A GB202003618D0 (en) | 2020-03-12 | 2020-03-12 | Gene Therapy |
| GB2003618.2 | 2020-03-12 | ||
| PCT/GB2021/050633 WO2021181118A1 (en) | 2020-03-12 | 2021-03-12 | Gene therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3171232A1 true CA3171232A1 (en) | 2021-09-16 |
Family
ID=70453740
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3171232A Pending CA3171232A1 (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 (2) | CN121950927A (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 |
| GB202309909D0 (en) * | 2023-06-29 | 2023-08-16 | Purespring Therapeutics Ltd | Regulatory elements |
| GB202309908D0 (en) * | 2023-06-29 | 2023-08-16 | Purespring Therapeutics Ltd | Polynucleotides, aav vectors, and pharmaceutical compositions for treating complement-mediated kidney diseases |
| GB202309913D0 (en) * | 2023-06-29 | 2023-08-16 | Purespring Therapeutics Ltd | Polynucleotides, avv vectors, and pharmaceutical compositions for treating nephrotic syndrome |
| GB202309914D0 (en) * | 2023-06-29 | 2023-08-16 | Purespring Therapeutics Ltd | Promoters |
| 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 |
|---|---|---|---|---|
| PT904392E (pt) | 1996-10-17 | 2001-06-29 | Oxford Biomedica Ltd | Vectores retrovirais |
| 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 |
| US7745389B2 (en) | 2005-02-14 | 2010-06-29 | University Of Iowa Research Foundation | Methods for treatment 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 |
| KR101105125B1 (ko) | 2007-05-07 | 2012-01-16 | 울산대학교 산학협력단 | 클러스터린을 이용한 체중 조절 장애 관련 질환의 진단, 예방 또는 치료 방법 |
| EP2730282A1 (en) | 2007-11-08 | 2014-05-14 | The General Hospital Corporation | Methods and compositions for the treatment of proteinuric diseases |
| US9279103B2 (en) | 2010-08-05 | 2016-03-08 | 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 |
| WO2016014781A1 (en) | 2014-07-25 | 2016-01-28 | Rgdi3, Inc. | Collagen iv replacement |
| AU2017341849B2 (en) * | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid 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 |
| CN118581154A (zh) | 2017-06-02 | 2024-09-03 | 国家健康与医学研究院 | 用于遗传障碍的基因疗法的基因组编辑手段和结合病毒载体的基因疗法 |
| EP3697920A4 (en) | 2017-10-20 | 2022-03-02 | Gemini Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF AGE-RELATED MACULAR DEGENERATION |
| RU2675861C1 (ru) | 2017-11-22 | 2018-12-25 | Лев Иосифович Шагам | Способ прогнозирования наличия мутации гена col4a5 у пациентов с предполагаемым диагнозом "синдром альпорта" |
| AU2019346335B2 (en) * | 2018-09-28 | 2024-07-25 | Massachusetts Institute Of Technology | Collagen-localized immunomodulatory molecules and methods thereof |
| CN113166208B (zh) * | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| 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 EP EP21711944.5A patent/EP4118216A1/en active Pending
- 2021-03-12 CN CN202511847212.8A patent/CN121950927A/zh active Pending
- 2021-03-12 CN CN202180020240.6A patent/CN115427578B/zh 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
- 2021-03-12 IL IL296255A patent/IL296255A/en unknown
- 2021-03-12 CA CA3171232A patent/CA3171232A1/en active Pending
- 2021-03-12 AU AU2021234169A patent/AU2021234169A1/en active Pending
- 2021-03-12 US US17/909,839 patent/US12589167B2/en active Active
- 2021-03-12 BR BR112022018036A patent/BR112022018036A2/pt unknown
- 2021-03-12 MX MX2022011295A patent/MX2022011295A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| US12589167B2 (en) | 2026-03-31 |
| EP4118216A1 (en) | 2023-01-18 |
| CN121950927A (zh) | 2026-05-01 |
| GB202003618D0 (en) | 2020-04-29 |
| AU2021234169A1 (en) | 2022-10-06 |
| IL296255A (en) | 2022-11-01 |
| BR112022018036A2 (pt) | 2022-11-01 |
| WO2021181118A1 (en) | 2021-09-16 |
| MX2022011295A (es) | 2022-11-14 |
| US20240197917A1 (en) | 2024-06-20 |
| JP2023517934A (ja) | 2023-04-27 |
| CN115427578B (zh) | 2026-03-17 |
| CN115427578A (zh) | 2022-12-02 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US12589167B2 (en) | AAV gene therapy vector with podocyte-specific promoter | |
| KR20180012772A (ko) | 파브리 병 유전자 치료 | |
| JP2022547197A (ja) | アデノ随伴ウイルス（ａａｖ）の生体内分布の変更に向けた、ａａｖとａａｖ受容体（ａａｖｒ）との間の相互作用を調節するための方法および組成物 | |
| CN103562396A (zh) | 用于无脉络膜的基因疗法的aav载体 | |
| US11891616B2 (en) | Transgene cassettes designed to express a human MECP2 gene | |
| WO2023278305A1 (en) | Methods and compositions for tau reduction gene therapy | |
| JP7820422B2 (ja) | ジスフェリン二重ベクターを用いた遺伝子治療 | |
| BR112021003174A2 (pt) | terapia gênica para o tratamento de galactosemia | |
| CN115715185A (zh) | 用于治疗神经系统疾病的组合物和方法 | |
| US20240376493A1 (en) | Optimized ap4m1 polyneucleotides and expression cassettes and their use | |
| CA3130221A1 (en) | Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd) | |
| JP7788594B2 (ja) | 乳児バッテン病の治療のための髄腔内および静脈内組合せ遺伝子療法 | |
| EP4410988A1 (en) | An aav2-vector variant for targeted transfer of genes | |
| AU2022274162A1 (en) | Vector system | |
| EP4615492A1 (en) | Polynucleotide and vector | |
| WO2025168940A1 (en) | Vectors | |
| WO2025003710A1 (en) | Polynucleotides, aav vectors, and pharmaceutical compositions for treating nephrotic syndrome | |
| WO2025003704A2 (en) | Polynucleotides, aav vectors, and pharmaceutical compositions for treating complement-mediated kidney diseases | |
| WO2025193938A1 (en) | Delivery of type iv collagen alpha chain using a split intein dual aav vector | |
| WO2023102518A1 (en) | Gnao1 gene therapy vectors and uses thereof |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| D11 | Substantive examination requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED Effective date: 20250228 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W200 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST OR RESPONSE SUBMITTED ONLINE Effective date: 20250228 Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20250228 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 4TH ANNIV.) - STANDARD Year of fee payment: 4 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20250307 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT Effective date: 20250307 Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20250307 |
|
| D00 | Search and/or examination requested or commenced |
Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D00-D118 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION REQUIREMENTS DETERMINED COMPLIANT Effective date: 20250310 |
|
| D11 | Substantive examination requested |
Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-D155 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALL REQUIREMENTS FOR EXAMINATION DETERMINED COMPLIANT Effective date: 20250429 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20250512 |
|
| R00 | Party data change recorded |
Free format text: ST27 STATUS EVENT CODE: A-2-2-R10-R00-R100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: SMALL ENTITY DECLARATION REQUEST RECEIVED Effective date: 20251216 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20251216 |
|
| R00 | Party data change recorded |
Free format text: ST27 STATUS EVENT CODE: A-2-2-R10-R00-R101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: SMALL ENTITY DECLARATION DETERMINED COMPLIANT Effective date: 20251219 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20251219 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 5TH ANNIV.) - SMALL Year of fee payment: 5 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20260302 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20260302 |