JP2022513318A - プロピオン酸血症を処置するための遺伝子治療 - Google Patents

プロピオン酸血症を処置するための遺伝子治療 Download PDF

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JP2022513318A
JP2022513318A JP2021542078A JP2021542078A JP2022513318A JP 2022513318 A JP2022513318 A JP 2022513318A JP 2021542078 A JP2021542078 A JP 2021542078A JP 2021542078 A JP2021542078 A JP 2021542078A JP 2022513318 A JP2022513318 A JP 2022513318A
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raav
pcca
seq
pccb
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JPWO2020072451A5 (https=
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マシュー スコット フラー，
サミュエル ウォズワース，
ケリー リード クラーク，
ショーン クリストファー ドーハティ，
スチュワート クレイグ，
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ウルトラジェニックス ファーマシューティカル インコーポレイテッド
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    • A—HUMAN NECESSITIES
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    • 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
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y604/00—Ligases forming carbon-carbon bonds (6.4)
    • C12Y604/01—Ligases forming carbon-carbon bonds (6.4.1)
    • C12Y604/01003—Propionyl-CoA carboxylase (6.4.1.3)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
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    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material

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  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2021542078A 2018-10-01 2019-10-01 プロピオン酸血症を処置するための遺伝子治療 Pending JP2022513318A (ja)

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US201862739471P 2018-10-01 2018-10-01
US62/739,471 2018-10-01
PCT/US2019/054003 WO2020072451A1 (en) 2018-10-01 2019-10-01 Gene therapy for treating propionic acidemia

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JP2022513318A true JP2022513318A (ja) 2022-02-07
JP2022513318A5 JP2022513318A5 (https=) 2022-10-12
JPWO2020072451A5 JPWO2020072451A5 (https=) 2022-10-12

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US (2) US12472268B2 (https=)
EP (1) EP3861131A4 (https=)
JP (1) JP2022513318A (https=)
KR (1) KR20210082460A (https=)
CN (1) CN112955557A (https=)
AR (1) AR116569A1 (https=)
AU (1) AU2019354296A1 (https=)
BR (1) BR112021006052A2 (https=)
CA (1) CA3113975A1 (https=)
TW (1) TW202028472A (https=)
WO (1) WO2020072451A1 (https=)

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EP3990029A1 (en) * 2019-06-27 2022-05-04 The United States of America, as represented by the Secretary, Department of Health and Human Services Synthetic genes for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase alpha
WO2021076797A1 (en) * 2019-10-15 2021-04-22 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Synthetic genes for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase beta
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
EP4308174A4 (en) * 2021-03-19 2025-07-30 Mayo Found Medical Education & Res METHODS AND MATERIALS FOR THE TREATMENT OF PROPIONIC ACIDEMIA
WO2023130003A2 (en) * 2021-12-29 2023-07-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Improved gene therapy constructs for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase alpha
CN118166004B (zh) * 2024-05-14 2024-12-03 北京剂泰医药科技有限公司 用于编码人PCCA或PCCB蛋白的mRNA及其用途

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US12472268B2 (en) 2025-11-18
KR20210082460A (ko) 2021-07-05
US20210283272A1 (en) 2021-09-16
AU2019354296A1 (en) 2021-04-29
WO2020072451A1 (en) 2020-04-09
TW202028472A (zh) 2020-08-01
EP3861131A1 (en) 2021-08-11
CN112955557A (zh) 2021-06-11
CA3113975A1 (en) 2020-04-09
EP3861131A4 (en) 2022-06-15
BR112021006052A2 (pt) 2021-09-08
WO2020072451A8 (en) 2021-05-14
AR116569A1 (es) 2021-05-19
US20260041788A1 (en) 2026-02-12

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