JP7042263B2 - ファブリー病の治療のための方法および組成物 - Google Patents

ファブリー病の治療のための方法および組成物 Download PDF

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JP7042263B2
JP7042263B2 JP2019520884A JP2019520884A JP7042263B2 JP 7042263 B2 JP7042263 B2 JP 7042263B2 JP 2019520884 A JP2019520884 A JP 2019520884A JP 2019520884 A JP2019520884 A JP 2019520884A JP 7042263 B2 JP7042263 B2 JP 7042263B2
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JP2020500162A (ja
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マーシャル ダブリュー． ヒューストン，
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Sangamo Therapeutics Inc
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    • 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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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
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    • 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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    • C12N2800/00—Nucleic acids vectors
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JP2019520884A 2016-10-20 2017-10-19 ファブリー病の治療のための方法および組成物 Active JP7042263B2 (ja)

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Application Number Priority Date Filing Date Title
JP2022039062A JP7418485B2 (ja) 2016-10-20 2022-03-14 ファブリー病の治療のための方法および組成物

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

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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)
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MX2021008131A (es) * 2019-01-04 2021-10-13 Sangamo Therapeutics Inc Metodos y composiciones para el tratamiento de la enfermedad de fabry.
PT3850089T (pt) 2019-02-04 2024-01-24 Freeline Therapeutics Ltd Polinucleótidos
EP3946433A4 (en) * 2019-03-27 2023-01-18 Sigilon Therapeutics, Inc. COMPOSITIONS, DEVICES AND METHODS FOR THE TREATMENT OF FABRY'S DISEASE
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
CN116802309A (zh) * 2020-07-15 2023-09-22 杰克逊实验室 用于sars-cov-2感染的人源化小鼠模型
WO2022155665A1 (en) * 2021-01-14 2022-07-21 Spark Therapeutics, Inc. Compositions and methods for treating fabry disease
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