JP7220080B2 - ハンチントン病治療組成物及び方法 - Google Patents

ハンチントン病治療組成物及び方法 Download PDF

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JP7220080B2
JP7220080B2 JP2018560628A JP2018560628A JP7220080B2 JP 7220080 B2 JP7220080 B2 JP 7220080B2 JP 2018560628 A JP2018560628 A JP 2018560628A JP 2018560628 A JP2018560628 A JP 2018560628A JP 7220080 B2 JP7220080 B2 JP 7220080B2
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seq
aav
htt
limiting example
expression
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JP2019519219A (ja
JP2019519219A5 (cg-RX-API-DMAC7.html
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ウェン-イー サー、ディナー
ホウ、ジンジャオ
ジョウ、ペンチェン
ワン、シン
デッカート、ヨヘン
ホスバッハ、マルクス
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Voyager Therapeutics Inc
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
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    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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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  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
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JP2018560628A 2016-05-18 2017-05-18 ハンチントン病治療組成物及び方法 Active JP7220080B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662338113P 2016-05-18 2016-05-18
US62/338,113 2016-05-18
US201762485049P 2017-04-13 2017-04-13
US62/485,049 2017-04-13
PCT/US2017/033281 WO2017201258A1 (en) 2016-05-18 2017-05-18 Compositions and methods of treating huntington's disease

Publications (3)

Publication Number Publication Date
JP2019519219A JP2019519219A (ja) 2019-07-11
JP2019519219A5 JP2019519219A5 (cg-RX-API-DMAC7.html) 2020-06-25
JP7220080B2 true JP7220080B2 (ja) 2023-02-09

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US (2) US11951121B2 (cg-RX-API-DMAC7.html)
EP (1) EP3458589A4 (cg-RX-API-DMAC7.html)
JP (1) JP7220080B2 (cg-RX-API-DMAC7.html)
KR (2) KR102427379B1 (cg-RX-API-DMAC7.html)
CN (1) CN109831916B (cg-RX-API-DMAC7.html)
AU (1) AU2017268382B2 (cg-RX-API-DMAC7.html)
BR (1) BR112018073472A2 (cg-RX-API-DMAC7.html)
CA (1) CA3024449A1 (cg-RX-API-DMAC7.html)
IL (2) IL297576B2 (cg-RX-API-DMAC7.html)
MX (1) MX2018014152A (cg-RX-API-DMAC7.html)
RU (1) RU2764587C2 (cg-RX-API-DMAC7.html)
SG (1) SG11201809643UA (cg-RX-API-DMAC7.html)
WO (1) WO2017201258A1 (cg-RX-API-DMAC7.html)

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