JP7066635B2 - 調節性ポリヌクレオチド - Google Patents

調節性ポリヌクレオチド Download PDF

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Publication number
JP7066635B2
JP7066635B2 JP2018560625A JP2018560625A JP7066635B2 JP 7066635 B2 JP7066635 B2 JP 7066635B2 JP 2018560625 A JP2018560625 A JP 2018560625A JP 2018560625 A JP2018560625 A JP 2018560625A JP 7066635 B2 JP7066635 B2 JP 7066635B2
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nucleic acid
acid sequence
sequence encoding
flanking region
region
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JP2019519218A5 (enExample
JP2019519218A (ja
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ウェン－イー サー、ディナー
ホウ、ジンジャオ
ノンネンマッチャー、マシュー
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Voyager Therapeutics Inc
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Voyager Therapeutics Inc
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Priority to JP2022072798A priority Critical patent/JP7374254B2/ja
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Priority to JP2023182415A priority patent/JP2023182824A/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • 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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    • C12N15/86—Viral vectors
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/50—Physical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Hospice & Palliative Care (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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JP2018560625A 2016-05-18 2017-05-18 調節性ポリヌクレオチド Active JP7066635B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022072798A JP7374254B2 (ja) 2016-05-18 2022-04-27 調節性ポリヌクレオチド
JP2023182415A JP2023182824A (ja) 2016-05-18 2023-10-24 調節性ポリヌクレオチド

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662338137P 2016-05-18 2016-05-18
US62/338,137 2016-05-18
US201762485050P 2017-04-13 2017-04-13
US62/485,050 2017-04-13
PCT/US2017/033268 WO2017201248A1 (en) 2016-05-18 2017-05-18 Modulatory polynucleotides

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JP2019519218A JP2019519218A (ja) 2019-07-11
JP2019519218A5 JP2019519218A5 (enExample) 2020-06-25
JP7066635B2 true JP7066635B2 (ja) 2022-05-13

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JP2022072798A Active JP7374254B2 (ja) 2016-05-18 2022-04-27 調節性ポリヌクレオチド
JP2023182415A Pending JP2023182824A (ja) 2016-05-18 2023-10-24 調節性ポリヌクレオチド

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US (3) US10584337B2 (enExample)
EP (1) EP3458588B1 (enExample)
JP (3) JP7066635B2 (enExample)
KR (3) KR102392236B1 (enExample)
CN (2) CN110214187B (enExample)
AU (2) AU2017267665C1 (enExample)
CA (2) CA3024448C (enExample)
IL (2) IL302748B1 (enExample)
MX (2) MX2018014154A (enExample)
RU (1) RU2758488C2 (enExample)
SG (1) SG11201809699XA (enExample)
WO (1) WO2017201248A1 (enExample)
ZA (1) ZA201807401B (enExample)

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