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DK3218386T3 - Modulatorisk polynukleotid - Google Patents

Modulatorisk polynukleotid Download PDF

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Publication number
DK3218386T3
DK3218386T3 DK15859587.6T DK15859587T DK3218386T3 DK 3218386 T3 DK3218386 T3 DK 3218386T3 DK 15859587 T DK15859587 T DK 15859587T DK 3218386 T3 DK3218386 T3 DK 3218386T3
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Prior art keywords
polynucleotid
modulatory
modulatory polynucleotid
Prior art date
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DK15859587.6T
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English (en)
Inventor
Jinzhao Hou
Mathieu E Nonnenmacher
Pengcheng Zhou
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Voyager Therapeutics Inc
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Publication of DK3218386T3 publication Critical patent/DK3218386T3/da

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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • 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
    • C12N15/1137—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 against enzymes
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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
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
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    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12N2330/00—Production
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
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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
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    • 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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  • 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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DK15859587.6T 2014-11-14 2015-11-13 Modulatorisk polynukleotid DK3218386T3 (da)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201462079590P 2014-11-14 2014-11-14
US201562212004P 2015-08-31 2015-08-31
US201562234477P 2015-09-29 2015-09-29
PCT/US2015/060564 WO2016077689A1 (en) 2014-11-14 2015-11-13 Modulatory polynucleotides

Publications (1)

Publication Number Publication Date
DK3218386T3 true DK3218386T3 (da) 2021-06-07

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DK15859587.6T DK3218386T3 (da) 2014-11-14 2015-11-13 Modulatorisk polynukleotid

Country Status (16)

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US (4) US10570395B2 (da)
EP (2) EP3218386B1 (da)
JP (3) JP6863891B2 (da)
KR (2) KR20230145206A (da)
CN (5) CN112410339A (da)
AU (3) AU2015346164B2 (da)
BR (1) BR112017010088A2 (da)
CA (1) CA2975583C (da)
DK (1) DK3218386T3 (da)
ES (1) ES2878451T3 (da)
IL (2) IL284949B2 (da)
MX (3) MX2017006217A (da)
RU (2) RU2719192C2 (da)
SG (2) SG10202001102XA (da)
WO (1) WO2016077689A1 (da)
ZA (1) ZA202004557B (da)

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