PE20210112A1 - Metodos de tratamiento de fenilcetonuria - Google Patents
Metodos de tratamiento de fenilcetonuriaInfo
- Publication number
- PE20210112A1 PE20210112A1 PE2020001818A PE2020001818A PE20210112A1 PE 20210112 A1 PE20210112 A1 PE 20210112A1 PE 2020001818 A PE2020001818 A PE 2020001818A PE 2020001818 A PE2020001818 A PE 2020001818A PE 20210112 A1 PE20210112 A1 PE 20210112A1
- Authority
- PE
- Peru
- Prior art keywords
- phenylketonuria
- methods
- treatment
- neurotransmitters
- normalizing
- 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
- C12N15/86—Viral vectors
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- 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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
- 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)
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- 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)
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/94—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving narcotics or drugs or pharmaceuticals, neurotransmitters or associated receptors
- G01N33/9406—Neurotransmitters
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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
- 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
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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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
- 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
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Abstract
En el presente documento se proporcionan metodos para tratar la fenilcetonuria normalizando los niveles de aminoacidos, neurotransmisores y metabolitos de neurotransmisores en un sujeto que tenga fenilcetonuria
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862669292P | 2018-05-09 | 2018-05-09 | |
US201862755207P | 2018-11-02 | 2018-11-02 | |
US201962802608P | 2019-02-07 | 2019-02-07 | |
US201962819414P | 2019-03-15 | 2019-03-15 | |
PCT/US2019/031252 WO2019217513A2 (en) | 2018-05-09 | 2019-05-08 | Methods of treating phenylketonuria |
Publications (1)
Publication Number | Publication Date |
---|---|
PE20210112A1 true PE20210112A1 (es) | 2021-01-19 |
Family
ID=66794083
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2020001818A PE20210112A1 (es) | 2018-05-09 | 2019-05-08 | Metodos de tratamiento de fenilcetonuria |
Country Status (15)
Country | Link |
---|---|
US (1) | US11739345B2 (es) |
EP (1) | EP3790627A2 (es) |
JP (1) | JP2021522811A (es) |
KR (1) | KR20210008501A (es) |
CN (1) | CN113164762A (es) |
AU (1) | AU2019265560A1 (es) |
BR (1) | BR112020022722A8 (es) |
CA (1) | CA3099704A1 (es) |
CL (1) | CL2020002892A1 (es) |
MX (1) | MX2020011937A (es) |
PE (1) | PE20210112A1 (es) |
PH (1) | PH12020551863A1 (es) |
SG (1) | SG11202010830WA (es) |
TW (1) | TW202016314A (es) |
WO (1) | WO2019217513A2 (es) |
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US20200318081A1 (en) * | 2017-10-02 | 2020-10-08 | American Gene Technologies International Inc. | Vectors with promoter and enhancer combinations for treating phenylketonuria |
JP7423521B2 (ja) | 2017-11-22 | 2024-01-29 | モダーナティエックス・インコーポレイテッド | フェニルケトン尿症の治療用のフェニルアラニンヒドロキシラーゼをコードするポリヌクレオチド |
TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
AU2021208972A1 (en) * | 2020-01-16 | 2022-08-11 | Takeda Pharmaceutical Company Limited | Adeno associated virus based gene therapy for phenylketonuria |
AR122409A1 (es) | 2020-04-03 | 2022-09-07 | Biomarin Pharm Inc | Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas |
WO2021207415A1 (en) * | 2020-04-07 | 2021-10-14 | The Curators Of The University Of Missouri | CpG-FREE ITRs FOR AAV GENE THERAPY |
WO2021252952A1 (en) * | 2020-06-11 | 2021-12-16 | Sangamo Therapeutics, Inc. | Methods and compositions for expressing phenylalanine hydroxylase |
MX2023005041A (es) | 2020-11-02 | 2023-05-17 | Biomarin Pharm Inc | Proceso para enriquecer virus adenoasociado. |
US20220160728A1 (en) * | 2020-11-05 | 2022-05-26 | Homology Medicines, Inc. | Gene therapy methods |
WO2023034980A1 (en) | 2021-09-03 | 2023-03-09 | Bomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034994A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034990A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034997A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034996A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034989A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
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 |
WO2024064856A1 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of cardiomyopathy with aav gene therapy vectors |
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2019
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- 2019-05-08 MX MX2020011937A patent/MX2020011937A/es unknown
- 2019-05-08 PE PE2020001818A patent/PE20210112A1/es unknown
- 2019-05-08 TW TW108115952A patent/TW202016314A/zh unknown
- 2019-05-08 CN CN201980046511.8A patent/CN113164762A/zh active Pending
- 2019-05-08 WO PCT/US2019/031252 patent/WO2019217513A2/en unknown
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- 2019-05-08 SG SG11202010830WA patent/SG11202010830WA/en unknown
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JP2021522811A (ja) | 2021-09-02 |
BR112020022722A8 (pt) | 2022-01-18 |
CA3099704A1 (en) | 2019-11-14 |
US11739345B2 (en) | 2023-08-29 |
PH12020551863A1 (en) | 2021-07-19 |
KR20210008501A (ko) | 2021-01-22 |
US20190376081A1 (en) | 2019-12-12 |
SG11202010830WA (en) | 2020-11-27 |
WO2019217513A2 (en) | 2019-11-14 |
CN113164762A (zh) | 2021-07-23 |
BR112020022722A2 (pt) | 2021-02-02 |
MX2020011937A (es) | 2021-01-29 |
WO2019217513A3 (en) | 2019-12-19 |
AU2019265560A1 (en) | 2020-11-26 |
TW202016314A (zh) | 2020-05-01 |
CL2020002892A1 (es) | 2021-05-07 |
EP3790627A2 (en) | 2021-03-17 |
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