MX2021004213A - Generacion de pah humana mejorada para el tratamiento de pku grave mediante terapia de sustitucion de genes dirigida al higado. - Google Patents

Generacion de pah humana mejorada para el tratamiento de pku grave mediante terapia de sustitucion de genes dirigida al higado.

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Publication number
MX2021004213A
MX2021004213A MX2021004213A MX2021004213A MX2021004213A MX 2021004213 A MX2021004213 A MX 2021004213A MX 2021004213 A MX2021004213 A MX 2021004213A MX 2021004213 A MX2021004213 A MX 2021004213A MX 2021004213 A MX2021004213 A MX 2021004213A
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MX
Mexico
Prior art keywords
liver
generation
treatment
replacement therapy
gene replacement
Prior art date
Application number
MX2021004213A
Other languages
English (en)
Spanish (es)
Inventor
Sirkka Kyostio-Moore
Partha Manavalan
Original Assignee
Genzyme Corp
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Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of MX2021004213A publication Critical patent/MX2021004213A/es

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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0071Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44Oxidoreductases (1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44Oxidoreductases (1)
    • A61K38/443Oxidoreductases (1) acting on CH-OH groups as donors, e.g. glucose oxidase, lactate dehydrogenase (1.1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52Genes encoding for enzymes or proenzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y114/00Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16Oxidoreductases 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)
    • C12Y114/16001Phenylalanine 4-monooxygenase (1.14.16.1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
MX2021004213A 2018-10-12 2019-10-11 Generacion de pah humana mejorada para el tratamiento de pku grave mediante terapia de sustitucion de genes dirigida al higado. MX2021004213A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862744944P 2018-10-12 2018-10-12
PCT/US2019/055917 WO2020077250A1 (en) 2018-10-12 2019-10-11 Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy

Publications (1)

Publication Number Publication Date
MX2021004213A true MX2021004213A (es) 2021-07-16

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Application Number Title Priority Date Filing Date
MX2021004213A MX2021004213A (es) 2018-10-12 2019-10-11 Generacion de pah humana mejorada para el tratamiento de pku grave mediante terapia de sustitucion de genes dirigida al higado.
MX2025013445A MX2025013445A (es) 2018-10-12 2021-04-12 Generacion de pah humana mejorada para el tratamiento de pku grave mediante terapia de sustitucion de genes dirigida al higado

Family Applications After (1)

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MX2025013445A MX2025013445A (es) 2018-10-12 2021-04-12 Generacion de pah humana mejorada para el tratamiento de pku grave mediante terapia de sustitucion de genes dirigida al higado

Country Status (16)

Country Link
US (1) US20210348135A1 (https=)
EP (2) EP3863662A1 (https=)
JP (2) JP2022504740A (https=)
KR (1) KR20210076051A (https=)
CN (2) CN120349985A (https=)
AR (1) AR116700A1 (https=)
AU (2) AU2019357039B2 (https=)
BR (1) BR112021006796A2 (https=)
CA (1) CA3116283A1 (https=)
CO (1) CO2021006060A2 (https=)
IL (1) IL282140A (https=)
MX (2) MX2021004213A (https=)
PH (1) PH12021550794A1 (https=)
SG (1) SG11202103614RA (https=)
TW (1) TW202035439A (https=)
WO (1) WO2020077250A1 (https=)

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AU2021261359A1 (en) * 2020-04-21 2022-12-08 Allen Institute Artificial expression constructs for selectively modulating gene expression in neocortical layer 5 glutamatergic neurons
JP2023524577A (ja) * 2020-05-05 2023-06-12 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 改変アデノ随伴ウイルス5カプシドおよびその使用
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
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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
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
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Publication number Publication date
BR112021006796A2 (pt) 2021-07-20
AU2019357039A1 (en) 2021-06-03
CN120349985A (zh) 2025-07-22
CN113194982B (zh) 2025-04-01
MX2025013445A (es) 2025-12-01
KR20210076051A (ko) 2021-06-23
AU2025237999A1 (en) 2025-10-23
EP4461814A2 (en) 2024-11-13
TW202035439A (zh) 2020-10-01
JP2022504740A (ja) 2022-01-13
SG11202103614RA (en) 2021-05-28
PH12021550794A1 (en) 2021-12-13
CA3116283A1 (en) 2020-04-16
CN113194982A (zh) 2021-07-30
EP3863662A1 (en) 2021-08-18
EP4461814A3 (en) 2025-02-26
AU2019357039B2 (en) 2025-10-09
JP2024123125A (ja) 2024-09-10
IL282140A (en) 2021-05-31
AR116700A1 (es) 2021-06-02
WO2020077250A1 (en) 2020-04-16
CO2021006060A2 (es) 2021-05-20
US20210348135A1 (en) 2021-11-11

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