BR112022026521A2 - Vetor viral, célula isolada, composição farmacêutica, uso de um vetor viral, método, e, método para prevenir ou tratar uma doença renal - Google Patents
Vetor viral, célula isolada, composição farmacêutica, uso de um vetor viral, método, e, método para prevenir ou tratar uma doença renalInfo
- Publication number
- BR112022026521A2 BR112022026521A2 BR112022026521A BR112022026521A BR112022026521A2 BR 112022026521 A2 BR112022026521 A2 BR 112022026521A2 BR 112022026521 A BR112022026521 A BR 112022026521A BR 112022026521 A BR112022026521 A BR 112022026521A BR 112022026521 A2 BR112022026521 A2 BR 112022026521A2
- Authority
- BR
- Brazil
- Prior art keywords
- viral vector
- treat
- pharmaceutical composition
- prevent
- kidney disease
- Prior art date
Links
- 239000013603 viral vector Substances 0.000 title abstract 6
- -1 ISOLATED CELL Substances 0.000 title abstract 2
- 208000017169 kidney disease Diseases 0.000 title abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 title abstract 2
- 239000002773 nucleotide Substances 0.000 abstract 2
- 125000003729 nucleotide group Chemical group 0.000 abstract 2
- 210000000557 podocyte Anatomy 0.000 abstract 2
- 102000000989 Complement System Proteins Human genes 0.000 abstract 1
- 108010069112 Complement System Proteins Proteins 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 230000002463 transducing 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
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Urology & Nephrology (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Ultra Sonic Daignosis Equipment (AREA)
- Image Analysis (AREA)
- Devices For Executing Special Programs (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB2010009.5A GB202010009D0 (en) | 2020-06-30 | 2020-06-30 | Vector |
| PCT/GB2021/051668 WO2022003357A1 (en) | 2020-06-30 | 2021-06-30 | Vector |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022026521A2 true BR112022026521A2 (pt) | 2023-01-31 |
Family
ID=71949697
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022026521A BR112022026521A2 (pt) | 2020-06-30 | 2021-06-30 | Vetor viral, célula isolada, composição farmacêutica, uso de um vetor viral, método, e, método para prevenir ou tratar uma doença renal |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20230175016A1 (https=) |
| EP (1) | EP4172345A1 (https=) |
| JP (1) | JP2023536008A (https=) |
| KR (1) | KR20230056658A (https=) |
| CN (1) | CN115997025A (https=) |
| AU (1) | AU2021300616A1 (https=) |
| BR (1) | BR112022026521A2 (https=) |
| CA (1) | CA3183830A1 (https=) |
| GB (1) | GB202010009D0 (https=) |
| IL (1) | IL299505A (https=) |
| MX (1) | MX2022016101A (https=) |
| WO (1) | WO2022003357A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB202003618D0 (en) | 2020-03-12 | 2020-04-29 | Univ Bristol | Gene Therapy |
| AU2021293174A1 (en) | 2020-06-14 | 2023-01-19 | Vertex Pharmaceuticals Incorporated | Complement Factor I-related compositions and methods |
| GB202217332D0 (en) * | 2022-11-18 | 2023-01-04 | Univ Bristol | Methods |
| EP4410988A1 (en) * | 2023-01-31 | 2024-08-07 | Universitätsklinikum Hamburg-Eppendorf | An aav2-vector variant for targeted transfer of genes |
| WO2024178007A1 (en) * | 2023-02-21 | 2024-08-29 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Mafb promoter for gene therapy for diabetes |
| GB202304841D0 (en) * | 2023-03-31 | 2023-05-17 | Purespring Therapeutics Ltd | Promoter |
| AU2024262827A1 (en) * | 2023-04-27 | 2025-10-16 | Oregon Health & Science University | Kidney-targeting aav capsids and methods of use thereof |
| GB202414499D0 (en) * | 2024-10-02 | 2024-11-13 | Purespring Therapeutics Ltd | Methods |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL332875A1 (en) | 1996-10-17 | 1999-10-25 | Oxford Biomedica Ltd | Retroviral vectors |
| 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 |
| ES2457822T3 (es) * | 2007-11-08 | 2014-04-29 | The General Hospital Corporation | Procedimientos y composiciones de tratamiento de enfermedades proteinúricas |
| ES2857773T5 (es) * | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| CN108291216B (zh) * | 2015-09-24 | 2022-09-16 | 宾夕法尼亚州大学信托人 | 用于治疗补体介导的疾病的组合物和方法 |
| JP7222075B2 (ja) * | 2018-09-13 | 2023-02-14 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | Ｃ３糸球体症のモデルとしての補体因子ｈ遺伝子ノックアウトラット |
-
2020
- 2020-06-30 GB GBGB2010009.5A patent/GB202010009D0/en not_active Ceased
-
2021
- 2021-06-30 MX MX2022016101A patent/MX2022016101A/es unknown
- 2021-06-30 CA CA3183830A patent/CA3183830A1/en active Pending
- 2021-06-30 EP EP21748644.8A patent/EP4172345A1/en active Pending
- 2021-06-30 JP JP2023523681A patent/JP2023536008A/ja active Pending
- 2021-06-30 US US18/011,592 patent/US20230175016A1/en active Pending
- 2021-06-30 AU AU2021300616A patent/AU2021300616A1/en active Pending
- 2021-06-30 CN CN202180046691.7A patent/CN115997025A/zh active Pending
- 2021-06-30 IL IL299505A patent/IL299505A/en unknown
- 2021-06-30 WO PCT/GB2021/051668 patent/WO2022003357A1/en not_active Ceased
- 2021-06-30 KR KR1020237003243A patent/KR20230056658A/ko active Pending
- 2021-06-30 BR BR112022026521A patent/BR112022026521A2/pt unknown
Also Published As
| Publication number | Publication date |
|---|---|
| IL299505A (en) | 2023-02-01 |
| GB202010009D0 (en) | 2020-08-12 |
| CA3183830A1 (en) | 2022-01-06 |
| KR20230056658A (ko) | 2023-04-27 |
| MX2022016101A (es) | 2023-06-01 |
| EP4172345A1 (en) | 2023-05-03 |
| AU2021300616A1 (en) | 2023-01-19 |
| US20230175016A1 (en) | 2023-06-08 |
| WO2022003357A1 (en) | 2022-01-06 |
| JP2023536008A (ja) | 2023-08-22 |
| CN115997025A (zh) | 2023-04-21 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B25A | Requested transfer of rights approved |
Owner name: THE UNIVERSITY OF BRISTOL (GB) ; PURESPRING THERAPEUTICS LIMITED (GB) ; SYNCONA IP HOLDCO (3) LIMITED (GB) |
|
| B25A | Requested transfer of rights approved |
Owner name: THE UNIVERSITY OF BRISTOL (GB) ; SYNCONA IP HOLDCO (3) LIMITED (GB) |