KR102353847B1 - 안구인두 근이영양증(opmd)의 치료용 시약 및 이의 용도 - Google Patents

안구인두 근이영양증(opmd)의 치료용 시약 및 이의 용도 Download PDF

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KR102353847B1
KR102353847B1 KR1020187032992A KR20187032992A KR102353847B1 KR 102353847 B1 KR102353847 B1 KR 102353847B1 KR 1020187032992 A KR1020187032992 A KR 1020187032992A KR 20187032992 A KR20187032992 A KR 20187032992A KR 102353847 B1 KR102353847 B1 KR 102353847B1
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데이비드 수히
마이클 그레이엄
카푸씬느 트호예
알베르토 말레르바
조지 제이. 딕슨
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베니텍 바이오파마 리미티드
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/04—Drugs for disorders of the respiratory system for throat disorders
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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  • Gastroenterology & Hepatology (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020187032992A 2016-04-14 2017-04-13 안구인두 근이영양증(opmd)의 치료용 시약 및 이의 용도 Active KR102353847B1 (ko)

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US201662322745P 2016-04-14 2016-04-14
US62/322,745 2016-04-14
PCT/AU2017/050330 WO2017177277A1 (en) 2016-04-14 2017-04-13 Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof

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KR102353847B1 true KR102353847B1 (ko) 2022-01-21

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EP (1) EP3443091A4 (enExample)
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CN (1) CN109219659B (enExample)
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CA (1) CA3020754C (enExample)
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Publication number Priority date Publication date Assignee Title
EP3443091A4 (en) 2016-04-14 2019-11-27 Benitec Biopharma Limited REAGENTS FOR THE TREATMENT OF OCULOPHARYNGEAL MUSCLE DYSTROPHY (OPMD) AND USE THEREOF
US11142765B2 (en) * 2016-12-14 2021-10-12 Benitec Ip Holdings, Inc. Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof
MX2021004225A (es) * 2018-10-17 2021-05-27 Benitec Ip Holdings Inc Metodos para tratar distrofia muscular oculofaringea (dmof).

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SG11201808981XA (en) 2018-11-29
NZ747314A (en) 2022-07-29
US20200138849A1 (en) 2020-05-07
KR20180133500A (ko) 2018-12-14
EP3443091A1 (en) 2019-02-20
WO2017177277A1 (en) 2017-10-19
BR112018071186A8 (pt) 2023-03-28
CA3020754C (en) 2023-07-25
MX2018012545A (es) 2019-06-10
CN109219659B (zh) 2022-09-09
EP3443091A4 (en) 2019-11-27
ZA201807589B (en) 2023-02-22
IL262337A (en) 2018-11-29
CA3020754A1 (en) 2017-10-19
JP2019513395A (ja) 2019-05-30
RU2755544C2 (ru) 2021-09-17
AU2017250017A1 (en) 2018-11-08
BR112018071186A2 (pt) 2019-02-12
RU2018138271A3 (enExample) 2020-09-22
RU2018138271A (ru) 2020-05-14
JP7236195B2 (ja) 2023-03-09
US11234994B2 (en) 2022-02-01
CN109219659A (zh) 2019-01-15
AU2017250017B2 (en) 2022-12-22
IL262337B2 (en) 2023-06-01

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