CN110022904B - 用于治疗法布里病的方法和组合物 - Google Patents

用于治疗法布里病的方法和组合物 Download PDF

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CN110022904B
CN110022904B CN201780072051.7A CN201780072051A CN110022904B CN 110022904 B CN110022904 B CN 110022904B CN 201780072051 A CN201780072051 A CN 201780072051A CN 110022904 B CN110022904 B CN 110022904B
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CN110022904A (zh
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M·W·赫斯顿
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Sangamo Therapeutics Inc
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • 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
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/067—Hepatocytes
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
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    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
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    • C12N2510/00—Genetically modified cells
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    • 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
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    • 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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  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
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  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Cell Biology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
CN201780072051.7A 2016-10-20 2017-10-19 用于治疗法布里病的方法和组合物 Active CN110022904B (zh)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US201662410543P 2016-10-20 2016-10-20
US62/410,543 2016-10-20
US201762444093P 2017-01-09 2017-01-09
US62/444,093 2017-01-09
US201762458324P 2017-02-13 2017-02-13
US62/458,324 2017-02-13
US201762502058P 2017-05-05 2017-05-05
US62/502,058 2017-05-05
US201762516373P 2017-06-07 2017-06-07
US62/516,373 2017-06-07
US201762552792P 2017-08-31 2017-08-31
US62/552,792 2017-08-31
PCT/US2017/057328 WO2018075736A1 (en) 2016-10-20 2017-10-19 Methods and compositions for the treatment of fabry disease

Publications (2)

Publication Number Publication Date
CN110022904A CN110022904A (zh) 2019-07-16
CN110022904B true CN110022904B (zh) 2024-04-19

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US (2) US11219695B2 (enExample)
EP (2) EP3528852B1 (enExample)
JP (2) JP7042263B2 (enExample)
KR (1) KR102712926B1 (enExample)
CN (1) CN110022904B (enExample)
AU (1) AU2017345430B2 (enExample)
BR (1) BR112019007210A2 (enExample)
CA (1) CA3039673A1 (enExample)
ES (1) ES3047063T3 (enExample)
IL (1) IL266047B2 (enExample)
MX (1) MX2019004487A (enExample)
WO (1) WO2018075736A1 (enExample)

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WO2020023390A1 (en) * 2018-07-25 2020-01-30 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
TWI860300B (zh) * 2018-10-18 2024-11-01 美商英特利亞醫療公司 用於從白蛋白基因座表現轉殖基因的組成物及方法
CN113260701A (zh) 2018-10-18 2021-08-13 英特利亚治疗股份有限公司 用于表达因子ix的组合物和方法
BR112021012249A2 (pt) * 2019-01-04 2021-09-08 Sangamo Therapeutics, Inc. Métodos e composições para o tratamento da doença de fabry
DK3850089T5 (da) 2019-02-04 2024-08-19 Freeline Therapeutics Ltd Polynukleotider
MX2021011754A (es) * 2019-03-27 2021-10-22 Sigilon Therapeutics Inc Composiciones, dispositivos y metodos para el tratamiento de la enfermedad de fabry.
BR112021022722A2 (pt) 2019-06-07 2022-01-04 Regeneron Pharma Animal não humano, célula de animal não humana, genoma de animal não humano, gene de albumina animal não humana humanizada, vetor de alvejamento, método de avaliação da atividade de um reagente, e, método de otimização da atividade de um reagente
CN113058041B (zh) * 2020-08-27 2022-04-05 华东师范大学 一种用于治疗庞贝氏病的产品
WO2021183895A1 (en) * 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
KR102438536B1 (ko) * 2020-04-28 2022-09-05 케이비바이오메드 주식회사 경구용 핵산 분자 전달 복합체
CA3190255A1 (en) * 2020-07-15 2022-01-20 The Jackson Laboratory Humanized mouse models for sars-cov-2 infection
AU2022207185A1 (en) * 2021-01-14 2023-07-13 Spark Therapeutics, Inc. Compositions and methods for treating fabry disease
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