MX2021011037A - Vectores de adn no virales y usos de estos para expresar agentes terapéuticos de fenilalanina hidroxilasa (pah). - Google Patents
Vectores de adn no virales y usos de estos para expresar agentes terapéuticos de fenilalanina hidroxilasa (pah).Info
- Publication number
- MX2021011037A MX2021011037A MX2021011037A MX2021011037A MX2021011037A MX 2021011037 A MX2021011037 A MX 2021011037A MX 2021011037 A MX2021011037 A MX 2021011037A MX 2021011037 A MX2021011037 A MX 2021011037A MX 2021011037 A MX2021011037 A MX 2021011037A
- Authority
- MX
- Mexico
- Prior art keywords
- cedna vectors
- pah
- pah protein
- expression
- methods
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- 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
- A61K48/0016—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 nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
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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
-
- 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
- A61K48/005—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
-
- 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
-
- 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
-
- 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.)
- C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
- C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Urology & Nephrology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
- Enzymes And Modification Thereof (AREA)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962817771P | 2019-03-13 | 2019-03-13 | |
| US201962857514P | 2019-06-05 | 2019-06-05 | |
| PCT/US2020/022595 WO2020186150A2 (en) | 2019-03-13 | 2020-03-13 | Non-viral dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) therapeutics |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021011037A true MX2021011037A (es) | 2021-12-15 |
Family
ID=72426457
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021011037A MX2021011037A (es) | 2019-03-13 | 2020-03-13 | Vectores de adn no virales y usos de estos para expresar agentes terapéuticos de fenilalanina hidroxilasa (pah). |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US12415002B2 (https=) |
| EP (1) | EP3938515A4 (https=) |
| JP (2) | JP7583732B2 (https=) |
| KR (1) | KR20210149702A (https=) |
| CN (1) | CN113874508A (https=) |
| AU (1) | AU2020235121A1 (https=) |
| BR (1) | BR112021017852A2 (https=) |
| CA (1) | CA3133330A1 (https=) |
| IL (1) | IL286283A (https=) |
| MX (1) | MX2021011037A (https=) |
| SG (1) | SG11202109830TA (https=) |
| WO (1) | WO2020186150A2 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4165196A1 (en) * | 2020-06-11 | 2023-04-19 | Sangamo Therapeutics, Inc. | Methods and compositions for expressing phenylalanine hydroxylase |
| JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Ｄｎａ分子の組成物、その作製方法、及びその使用方法 |
| EP4214309A4 (en) * | 2020-09-16 | 2025-07-16 | Generation Bio Co | CLOSED-END DNA VECTORS AND RELATED USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) |
| GB202014751D0 (en) * | 2020-09-18 | 2020-11-04 | Lightbio Ltd | Targeting vector |
| MX2023003805A (es) | 2020-10-01 | 2023-04-12 | Genzyme Corp | Casete de expresion de pah humana para el tratamiento de la pku mediante terapia de reemplazo genico dirigida al higado. |
| CN115772503B (zh) * | 2022-08-16 | 2023-10-17 | 广东省南山医药创新研究院 | 一种表达pah的基因修饰细胞药物及其制备方法与应用 |
| AU2024257493A1 (en) * | 2023-04-18 | 2025-12-04 | Siren Biotechnology, Inc. | Adeno-associated viral vector compositions and methods of use |
| CN116656740A (zh) * | 2023-05-30 | 2023-08-29 | 浙江大学 | 一种腺相关病毒基因治疗载体及其在制备治疗苯丙酮尿症的药物中的应用 |
| CN119876192B (zh) * | 2024-07-02 | 2025-11-07 | 浙江大学滨江研究院 | 用于治疗苯丙酮尿症的多核苷酸 |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2535877T3 (es) | 2006-06-19 | 2015-05-18 | Asklepios Biopharmaceutical, Inc. | Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica |
| 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 |
| MX2018010633A (es) | 2016-03-03 | 2019-06-13 | Univ Massachusetts | Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral. |
| KR102738752B1 (ko) | 2016-12-30 | 2024-12-06 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 페닐케톤뇨증을 치료하기 위한 유전자 치료 |
| WO2018167621A1 (en) | 2017-03-16 | 2018-09-20 | Pfizer Inc. | Tyrosine prototrophy |
| US11820999B2 (en) * | 2017-04-03 | 2023-11-21 | American Gene Technologies International Inc. | Compositions and methods for treating phenylketonuria |
| EP3630200A4 (en) | 2017-05-31 | 2021-02-24 | Arcturus Therapeutics, Inc. | THERAPEUTICS FOR PHENYLKETONURIA |
| NZ762100A (en) * | 2017-08-09 | 2025-12-19 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof |
-
2020
- 2020-03-13 EP EP20768953.0A patent/EP3938515A4/en active Pending
- 2020-03-13 JP JP2021554695A patent/JP7583732B2/ja active Active
- 2020-03-13 US US17/437,172 patent/US12415002B2/en active Active
- 2020-03-13 MX MX2021011037A patent/MX2021011037A/es unknown
- 2020-03-13 BR BR112021017852A patent/BR112021017852A2/pt unknown
- 2020-03-13 AU AU2020235121A patent/AU2020235121A1/en active Pending
- 2020-03-13 CN CN202080035845.8A patent/CN113874508A/zh active Pending
- 2020-03-13 KR KR1020217029544A patent/KR20210149702A/ko active Pending
- 2020-03-13 WO PCT/US2020/022595 patent/WO2020186150A2/en not_active Ceased
- 2020-03-13 CA CA3133330A patent/CA3133330A1/en active Pending
- 2020-03-13 SG SG11202109830T patent/SG11202109830TA/en unknown
-
2021
- 2021-09-12 IL IL286283A patent/IL286283A/en unknown
-
2024
- 2024-11-01 JP JP2024193194A patent/JP2025016685A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| KR20210149702A (ko) | 2021-12-09 |
| EP3938515A2 (en) | 2022-01-19 |
| JP2022525302A (ja) | 2022-05-12 |
| CN113874508A (zh) | 2021-12-31 |
| CA3133330A1 (en) | 2020-09-17 |
| WO2020186150A3 (en) | 2020-10-29 |
| JP2025016685A (ja) | 2025-02-04 |
| EP3938515A4 (en) | 2022-12-28 |
| BR112021017852A2 (pt) | 2021-11-30 |
| JP7583732B2 (ja) | 2024-11-14 |
| IL286283A (en) | 2021-10-31 |
| SG11202109830TA (en) | 2021-10-28 |
| WO2020186150A2 (en) | 2020-09-17 |
| AU2020235121A1 (en) | 2021-11-04 |
| US12415002B2 (en) | 2025-09-16 |
| US20230024354A1 (en) | 2023-01-26 |
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