MX2020008676A - Expresion controlada de transgenes mediante el uso de vectores de adn de extremo cerrado (ceadn). - Google Patents
Expresion controlada de transgenes mediante el uso de vectores de adn de extremo cerrado (ceadn).Info
- Publication number
- MX2020008676A MX2020008676A MX2020008676A MX2020008676A MX2020008676A MX 2020008676 A MX2020008676 A MX 2020008676A MX 2020008676 A MX2020008676 A MX 2020008676A MX 2020008676 A MX2020008676 A MX 2020008676A MX 2020008676 A MX2020008676 A MX 2020008676A
- Authority
- MX
- Mexico
- Prior art keywords
- level
- expression
- transgene
- cedna
- close
- Prior art date
Links
- 108700019146 Transgenes Proteins 0.000 title abstract 7
- 239000013598 vector Substances 0.000 title abstract 3
- 238000000034 method Methods 0.000 abstract 2
- 230000037452 priming Effects 0.000 abstract 2
- 238000001415 gene therapy Methods 0.000 abstract 1
- 238000004448 titration Methods 0.000 abstract 1
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
- A61K48/0083—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 administration regime
-
- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
- A61P17/06—Antipsoriatics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/14011—Baculoviridae
- C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
- C12N2710/14141—Use of virus, viral particle or viral elements as a vector
- C12N2710/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
- C12N2750/14143—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
- C12N2800/50—Vectors for producing 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0069—Oxidoreductases (1.) acting on single donors with incorporation of molecular oxygen, i.e. oxygenases (1.13)
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)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Dermatology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Hospice & Palliative Care (AREA)
- Physical Education & Sports Medicine (AREA)
- Rheumatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Immunology (AREA)
- Psychiatry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862633757P | 2018-02-22 | 2018-02-22 | |
| US201862633795P | 2018-02-22 | 2018-02-22 | |
| US201862633882P | 2018-02-22 | 2018-02-22 | |
| US201862746762P | 2018-10-17 | 2018-10-17 | |
| PCT/US2019/018927 WO2019165050A1 (en) | 2018-02-22 | 2019-02-21 | Controlled expression of transgenes using close-ended dna (cedna) vectors |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2020008676A true MX2020008676A (es) | 2020-09-25 |
Family
ID=67687406
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2020008676A MX2020008676A (es) | 2018-02-22 | 2019-02-21 | Expresion controlada de transgenes mediante el uso de vectores de adn de extremo cerrado (ceadn). |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20220175970A1 (https=) |
| EP (1) | EP3755803A4 (https=) |
| JP (2) | JP2021513999A (https=) |
| KR (1) | KR20200124250A (https=) |
| CN (1) | CN111886343A (https=) |
| AU (1) | AU2019225937A1 (https=) |
| BR (1) | BR112020017060A2 (https=) |
| CA (1) | CA3092059A1 (https=) |
| IL (1) | IL276658A (https=) |
| MA (1) | MA51915A (https=) |
| MX (1) | MX2020008676A (https=) |
| PH (1) | PH12020551235A1 (https=) |
| SG (1) | SG11202007621TA (https=) |
| WO (1) | WO2019165050A1 (https=) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TW202545984A (zh) | 2017-08-09 | 2025-12-01 | 美商生物化學醫療公司 | 核酸分子及其用途 |
| WO2020033863A1 (en) | 2018-08-09 | 2020-02-13 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| CN113316640A (zh) * | 2018-11-09 | 2021-08-27 | 世代生物公司 | 包含对称的被修饰的反向末端重复序列的被修饰的封闭端dna(cedna) |
| DE102020111571A1 (de) | 2020-03-11 | 2021-09-16 | Immatics US, Inc. | Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren |
| AU2021244559A1 (en) * | 2020-03-24 | 2022-11-17 | Generation Bio Co. | Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics |
| MX2022011805A (es) * | 2020-03-24 | 2023-01-11 | Generation Bio Co | Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix. |
| JP2023524010A (ja) * | 2020-04-28 | 2023-06-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 高効率遺伝子送達系 |
| US20230265440A1 (en) * | 2020-06-26 | 2023-08-24 | The Board Of Trustees Of The Leland Stanford Junior University | Targeting the human ccr5 locus as a safe harbor for the expression of therapeutic proteins |
| CN116234917A (zh) | 2020-07-27 | 2023-06-06 | 安杰瑞姆生物科学公司 | Dna分子组合物及其制备方法和使用方法 |
| US20220090130A1 (en) * | 2020-08-23 | 2022-03-24 | Bioverativ Therapeutics Inc. | MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA) |
| US20240026374A1 (en) * | 2020-09-16 | 2024-01-25 | Generation Bio Co. | Closed-ended dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) |
| CA3211687A1 (en) * | 2021-03-19 | 2022-09-22 | Ozan ALKAN | Non-viral dna vectors and uses thereof for expressing pfic therapeutics |
| WO2025114932A1 (en) * | 2023-11-30 | 2025-06-05 | Sanofi | Purification of closed-ended dna molecules |
| CN121662213B (zh) * | 2026-02-06 | 2026-04-21 | 湖南博极生命科技有限公司 | 基于注意力权重追溯的中药活性成分可解释性分析方法及系统 |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003087334A2 (en) * | 2002-04-09 | 2003-10-23 | Cornell Research Foundation, Inc. | Use of aav integration efficiency element for mediating site-specific integration of a transcription unit |
| LT2816118T (lt) * | 2005-05-31 | 2018-12-10 | The Regents Of The University Of Colorado, A Body Corporate | Genų pristatymo būdai |
| JP2010538675A (ja) * | 2007-09-19 | 2010-12-16 | アムステルダム モレキュラー セラピューティクス ビー．ブイ． | タンパク質産生の改善のためのａａｖ複製機構の使用 |
| EP2500434A1 (en) * | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
| WO2015191508A1 (en) * | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| AU2015311704B2 (en) * | 2014-09-07 | 2021-12-09 | Selecta Biosciences, Inc. | Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses |
| EP3889260A1 (en) * | 2014-12-12 | 2021-10-06 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
| WO2017152149A1 (en) * | 2016-03-03 | 2017-09-08 | University Of Massachusetts | Closed-ended linear duplex dna for non-viral gene transfer |
-
2019
- 2019-02-21 MA MA051915A patent/MA51915A/fr unknown
- 2019-02-21 BR BR112020017060-7A patent/BR112020017060A2/pt not_active Application Discontinuation
- 2019-02-21 PH PH1/2020/551235A patent/PH12020551235A1/en unknown
- 2019-02-21 MX MX2020008676A patent/MX2020008676A/es unknown
- 2019-02-21 AU AU2019225937A patent/AU2019225937A1/en not_active Abandoned
- 2019-02-21 SG SG11202007621TA patent/SG11202007621TA/en unknown
- 2019-02-21 WO PCT/US2019/018927 patent/WO2019165050A1/en not_active Ceased
- 2019-02-21 KR KR1020207026566A patent/KR20200124250A/ko not_active Ceased
- 2019-02-21 US US16/971,016 patent/US20220175970A1/en active Pending
- 2019-02-21 CA CA3092059A patent/CA3092059A1/en active Pending
- 2019-02-21 EP EP19757005.4A patent/EP3755803A4/en active Pending
- 2019-02-21 CN CN201980019861.5A patent/CN111886343A/zh active Pending
- 2019-02-21 JP JP2020544445A patent/JP2021513999A/ja active Pending
-
2020
- 2020-08-11 IL IL276658A patent/IL276658A/en unknown
-
2023
- 2023-12-08 JP JP2023207938A patent/JP2024028931A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2024028931A (ja) | 2024-03-05 |
| BR112020017060A2 (pt) | 2020-12-15 |
| PH12020551235A1 (en) | 2021-04-19 |
| WO2019165050A1 (en) | 2019-08-29 |
| MA51915A (fr) | 2020-12-30 |
| EP3755803A1 (en) | 2020-12-30 |
| CA3092059A1 (en) | 2019-08-29 |
| US20220175970A1 (en) | 2022-06-09 |
| CN111886343A (zh) | 2020-11-03 |
| JP2021513999A (ja) | 2021-06-03 |
| SG11202007621TA (en) | 2020-09-29 |
| EP3755803A4 (en) | 2022-01-19 |
| KR20200124250A (ko) | 2020-11-02 |
| IL276658A (en) | 2020-09-30 |
| RU2020131041A (ru) | 2022-03-22 |
| AU2019225937A1 (en) | 2020-08-13 |
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