BR112020014191A2 - Unidade de controle transcricional (uct), construção de expressão, vetor, célula hospedeira, composição farmacêutica, uso de um vetor, método para tratamento ou prevenção de distúrbios da retina e vetores para uso - Google Patents
Unidade de controle transcricional (uct), construção de expressão, vetor, célula hospedeira, composição farmacêutica, uso de um vetor, método para tratamento ou prevenção de distúrbios da retina e vetores para usoInfo
- Publication number
- BR112020014191A2 BR112020014191A2 BR112020014191A BR112020014191A BR112020014191A2 BR 112020014191 A2 BR112020014191 A2 BR 112020014191A2 BR 112020014191 A BR112020014191 A BR 112020014191A BR 112020014191 A BR112020014191 A BR 112020014191A BR 112020014191 A2 BR112020014191 A2 BR 112020014191A2
- Authority
- BR
- Brazil
- Prior art keywords
- vector
- uct
- vectors
- treating
- control unit
- Prior art date
Links
Classifications
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- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
- A61K39/235—Adenoviridae
-
- 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
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
-
- 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
- A61P27/02—Ophthalmic agents
-
- 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
- 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
- C12N15/861—Adenoviral 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
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Virology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Ophthalmology & Optometry (AREA)
- Mycology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Immunology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1800546.2A GB201800546D0 (en) | 2018-01-12 | 2018-01-12 | Treatment |
| PCT/GB2019/050092 WO2019138250A1 (en) | 2018-01-12 | 2019-01-14 | Compositions and methods for treating retinal disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112020014191A2 true BR112020014191A2 (pt) | 2022-02-22 |
Family
ID=61256183
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112020014191A BR112020014191A2 (pt) | 2018-01-12 | 2019-01-14 | Unidade de controle transcricional (uct), construção de expressão, vetor, célula hospedeira, composição farmacêutica, uso de um vetor, método para tratamento ou prevenção de distúrbios da retina e vetores para uso |
Country Status (26)
| Country | Link |
|---|---|
| US (2) | US11680276B2 (https=) |
| EP (1) | EP3737423B1 (https=) |
| JP (1) | JP7289306B2 (https=) |
| KR (1) | KR102786552B1 (https=) |
| CN (1) | CN111936172A (https=) |
| AU (1) | AU2019206314B2 (https=) |
| BR (1) | BR112020014191A2 (https=) |
| CA (1) | CA3088079A1 (https=) |
| DK (1) | DK3737423T3 (https=) |
| EA (1) | EA202091655A1 (https=) |
| ES (1) | ES2988499T3 (https=) |
| FI (1) | FI3737423T3 (https=) |
| GB (1) | GB201800546D0 (https=) |
| HR (1) | HRP20241483T1 (https=) |
| HU (1) | HUE068987T2 (https=) |
| IL (1) | IL275985A (https=) |
| LT (1) | LT3737423T (https=) |
| MX (1) | MX2020007366A (https=) |
| PH (1) | PH12020551071A1 (https=) |
| PL (1) | PL3737423T3 (https=) |
| PT (1) | PT3737423T (https=) |
| RS (1) | RS66227B1 (https=) |
| SG (1) | SG11202006618PA (https=) |
| SI (1) | SI3737423T1 (https=) |
| SM (1) | SMT202400410T1 (https=) |
| WO (1) | WO2019138250A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11241460B2 (en) | 2013-03-15 | 2022-02-08 | Astellas Institute For Regenerative Medicine | Photoreceptors and photoreceptor progenitors produced from pluripotent stem cells |
| EP4292600A3 (en) | 2013-03-15 | 2024-04-10 | Astellas Institute for Regenerative Medicine | Photoreceptors and photoreceptor progenitors produced from pluripotent stem cells |
| GB2584664B (en) * | 2019-06-10 | 2023-05-24 | Newcells Biotech Ltd | Improved retinal organoids and methods of making the same |
| CN113025616B (zh) * | 2021-04-21 | 2023-05-02 | 中国热带农业科学院橡胶研究所 | 一种橡胶树泛素基因启动子proHbUBI2及其克隆与应用 |
| AU2022275948A1 (en) * | 2021-05-20 | 2023-12-21 | Artema Therapeutics, Inc. | Kcnv2 variants and their use |
| WO2023039175A2 (en) * | 2021-09-09 | 2023-03-16 | Applied Genetic Technologies Corporation | Predictors of achromatopsia treatment efficacy |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008073303A2 (en) * | 2006-12-07 | 2008-06-19 | Switchgear Genomics | Transcriptional regulatory elements of biological pathways, tools, and methods |
| US20120172419A1 (en) | 2009-09-15 | 2012-07-05 | Medical College Of Wisconsin Research Foundation Inc. | Reagents and methods for modulating cone photoreceptor activity |
| CN103608455B (zh) * | 2011-01-07 | 2019-01-15 | 应用遗传科技公司 | 用于治疗色盲和其它疾病的启动子、表达盒、载体、药盒和方法 |
| EP3800191B1 (en) * | 2014-03-17 | 2025-08-20 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| US11021519B2 (en) * | 2015-03-02 | 2021-06-01 | Adverum Biotechnologies, Inc. | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| WO2017144080A1 (en) * | 2016-02-23 | 2017-08-31 | Eyeserv Gmbh | Gene therapy for the treatment of a disease of retinal cone cells |
-
2018
- 2018-01-12 GB GBGB1800546.2A patent/GB201800546D0/en not_active Ceased
-
2019
- 2019-01-14 CA CA3088079A patent/CA3088079A1/en active Pending
- 2019-01-14 EP EP19701018.4A patent/EP3737423B1/en active Active
- 2019-01-14 HR HRP20241483TT patent/HRP20241483T1/hr unknown
- 2019-01-14 PL PL19701018.4T patent/PL3737423T3/pl unknown
- 2019-01-14 EA EA202091655A patent/EA202091655A1/ru unknown
- 2019-01-14 HU HUE19701018A patent/HUE068987T2/hu unknown
- 2019-01-14 CN CN201980018443.4A patent/CN111936172A/zh active Pending
- 2019-01-14 JP JP2020538584A patent/JP7289306B2/ja active Active
- 2019-01-14 KR KR1020207023349A patent/KR102786552B1/ko active Active
- 2019-01-14 PT PT197010184T patent/PT3737423T/pt unknown
- 2019-01-14 RS RS20241186A patent/RS66227B1/sr unknown
- 2019-01-14 SI SI201930833T patent/SI3737423T1/sl unknown
- 2019-01-14 BR BR112020014191A patent/BR112020014191A2/pt unknown
- 2019-01-14 ES ES19701018T patent/ES2988499T3/es active Active
- 2019-01-14 SM SM20240410T patent/SMT202400410T1/it unknown
- 2019-01-14 US US16/961,857 patent/US11680276B2/en active Active
- 2019-01-14 SG SG11202006618PA patent/SG11202006618PA/en unknown
- 2019-01-14 MX MX2020007366A patent/MX2020007366A/es unknown
- 2019-01-14 DK DK19701018.4T patent/DK3737423T3/da active
- 2019-01-14 LT LTEPPCT/GB2019/050092T patent/LT3737423T/lt unknown
- 2019-01-14 WO PCT/GB2019/050092 patent/WO2019138250A1/en not_active Ceased
- 2019-01-14 FI FIEP19701018.4T patent/FI3737423T3/fi active
- 2019-01-14 AU AU2019206314A patent/AU2019206314B2/en active Active
-
2020
- 2020-07-10 PH PH12020551071A patent/PH12020551071A1/en unknown
- 2020-07-12 IL IL275985A patent/IL275985A/en unknown
-
2023
- 2023-06-16 US US18/336,409 patent/US12467066B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| SG11202006618PA (en) | 2020-08-28 |
| SMT202400410T1 (it) | 2025-01-14 |
| SI3737423T1 (sl) | 2025-03-31 |
| EP3737423B1 (en) | 2024-09-18 |
| ES2988499T3 (es) | 2024-11-20 |
| JP2021510301A (ja) | 2021-04-22 |
| US11680276B2 (en) | 2023-06-20 |
| NZ766316A (en) | 2024-05-31 |
| PT3737423T (pt) | 2024-10-03 |
| PH12020551071A1 (en) | 2021-09-06 |
| EA202091655A1 (ru) | 2020-11-20 |
| LT3737423T (lt) | 2024-11-25 |
| HUE068987T2 (hu) | 2025-02-28 |
| AU2019206314A1 (en) | 2020-08-13 |
| KR20200141435A (ko) | 2020-12-18 |
| US20200392536A1 (en) | 2020-12-17 |
| DK3737423T3 (da) | 2024-10-21 |
| PL3737423T3 (pl) | 2025-01-27 |
| HRP20241483T1 (hr) | 2025-01-03 |
| GB201800546D0 (en) | 2018-02-28 |
| CN111936172A (zh) | 2020-11-13 |
| MX2020007366A (es) | 2020-09-03 |
| JP7289306B2 (ja) | 2023-06-09 |
| IL275985A (en) | 2020-08-31 |
| WO2019138250A1 (en) | 2019-07-18 |
| US12467066B2 (en) | 2025-11-11 |
| EP3737423A1 (en) | 2020-11-18 |
| FI3737423T3 (fi) | 2024-11-02 |
| CA3088079A1 (en) | 2019-07-18 |
| US20240067989A1 (en) | 2024-02-29 |
| RS66227B1 (sr) | 2024-12-31 |
| KR102786552B1 (ko) | 2025-03-26 |
| AU2019206314B2 (en) | 2025-02-13 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B06W | Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette] |