SG11202109830TA - Non-viral dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) therapeutics - Google Patents
Non-viral dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) therapeuticsInfo
- Publication number
- SG11202109830TA SG11202109830TA SG11202109830TA SG11202109830TA SG 11202109830T A SG11202109830T A SG 11202109830TA SG 11202109830T A SG11202109830T A SG 11202109830TA SG 11202109830T A SG11202109830T A SG 11202109830TA
- Authority
- SG
- Singapore
- Prior art keywords
- pah
- therapeutics
- viral dna
- dna vectors
- phenylalanine hydroxylase
- 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
-
- 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)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (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)
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 |
---|---|
SG11202109830TA true SG11202109830TA (en) | 2021-10-28 |
Family
ID=72426457
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11202109830T SG11202109830TA (en) | 2019-03-13 | 2020-03-13 | Non-viral dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) therapeutics |
Country Status (12)
Country | Link |
---|---|
US (1) | US20230024354A1 (de) |
EP (1) | EP3938515A4 (de) |
JP (1) | JP2022525302A (de) |
KR (1) | KR20210149702A (de) |
CN (1) | CN113874508A (de) |
AU (1) | AU2020235121A1 (de) |
BR (1) | BR112021017852A2 (de) |
CA (1) | CA3133330A1 (de) |
IL (1) | IL286283A (de) |
MX (1) | MX2021011037A (de) |
SG (1) | SG11202109830TA (de) |
WO (1) | WO2020186150A2 (de) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
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 |
KR20230066615A (ko) * | 2020-09-16 | 2023-05-16 | 제너레이션 바이오 컴퍼니 | 폐쇄형 dna 벡터 및 페닐알라닌 히드록실라아제(pah) 발현을 위한 이의 용도 |
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 |
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 |
Family Cites Families (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP2037892B1 (de) * | 2006-06-19 | 2015-03-18 | Asklepios Biopharmaceutical, Inc. | Modifizierte fatkor-viii- und faktor-ix-gene und vektoren für gentherapie |
SG11201806663TA (en) * | 2016-03-03 | 2018-09-27 | Univ Massachusetts | Closed-ended linear duplex dna for non-viral gene transfer |
KR20190100318A (ko) * | 2016-12-30 | 2019-08-28 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 페닐케톤뇨증을 치료하기 위한 유전자 치료 |
US20200056190A1 (en) * | 2017-03-16 | 2020-02-20 | Pfizer Inc. | Tyrosine prototrophy |
CA3057142A1 (en) * | 2017-04-03 | 2018-10-11 | American Gene Technologies International Inc. | Compositions and methods for treating phenylketonuria |
WO2018222925A1 (en) * | 2017-05-31 | 2018-12-06 | Ultragenyx Pharmaceutical Inc. | Therapeutics for phenylketonuria |
EP3665289A1 (de) * | 2017-08-09 | 2020-06-17 | Bioverativ Therapeutics Inc. | Nukleinsäuremoleküle und verwendungen davon |
-
2020
- 2020-03-13 KR KR1020217029544A patent/KR20210149702A/ko 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 CA CA3133330A patent/CA3133330A1/en active Pending
- 2020-03-13 US US17/437,172 patent/US20230024354A1/en active Pending
- 2020-03-13 EP EP20768953.0A patent/EP3938515A4/de active Pending
- 2020-03-13 MX MX2021011037A patent/MX2021011037A/es unknown
- 2020-03-13 SG SG11202109830T patent/SG11202109830TA/en unknown
- 2020-03-13 WO PCT/US2020/022595 patent/WO2020186150A2/en unknown
- 2020-03-13 BR BR112021017852A patent/BR112021017852A2/pt unknown
- 2020-03-13 JP JP2021554695A patent/JP2022525302A/ja active Pending
-
2021
- 2021-09-12 IL IL286283A patent/IL286283A/en unknown
Also Published As
Publication number | Publication date |
---|---|
AU2020235121A1 (en) | 2021-11-04 |
EP3938515A2 (de) | 2022-01-19 |
CA3133330A1 (en) | 2020-09-17 |
KR20210149702A (ko) | 2021-12-09 |
EP3938515A4 (de) | 2022-12-28 |
IL286283A (en) | 2021-10-31 |
US20230024354A1 (en) | 2023-01-26 |
MX2021011037A (es) | 2021-12-15 |
WO2020186150A3 (en) | 2020-10-29 |
BR112021017852A2 (pt) | 2021-11-30 |
JP2022525302A (ja) | 2022-05-12 |
WO2020186150A2 (en) | 2020-09-17 |
CN113874508A (zh) | 2021-12-31 |
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