AU2017377661B2 - Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof - Google Patents

Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof Download PDF

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AU2017377661B2
AU2017377661B2 AU2017377661A AU2017377661A AU2017377661B2 AU 2017377661 B2 AU2017377661 B2 AU 2017377661B2 AU 2017377661 A AU2017377661 A AU 2017377661A AU 2017377661 A AU2017377661 A AU 2017377661A AU 2017377661 B2 AU2017377661 B2 AU 2017377661B2
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set forth
nucleic acid
shmir
effector
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AU2017377661A1 (en
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Vanessa STRINGS-UFOMBAH
David Suhy
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Benitec IP Holdings Inc
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    • 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
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
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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
    • C12N15/113—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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    • C12N15/00—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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/861—Adenoviral vectors
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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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/867—Retroviral 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]
    • C—CHEMISTRY; METALLURGY
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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]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2750/14141—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Neurology (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)
  • Investigating Or Analysing Biological Materials (AREA)
AU2017377661A 2016-12-14 2017-12-14 Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof Active AU2017377661B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2023222827A AU2023222827B2 (en) 2016-12-14 2023-08-28 Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662434312P 2016-12-14 2016-12-14
US62/434,312 2016-12-14
PCT/AU2017/051385 WO2018107228A1 (en) 2016-12-14 2017-12-14 Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof

Related Child Applications (1)

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AU2023222827A Division AU2023222827B2 (en) 2016-12-14 2023-08-28 Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof

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AU2017377661A1 AU2017377661A1 (en) 2019-07-18
AU2017377661B2 true AU2017377661B2 (en) 2023-07-27

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AU2017377661A Active AU2017377661B2 (en) 2016-12-14 2017-12-14 Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof
AU2023222827A Active AU2023222827B2 (en) 2016-12-14 2023-08-28 Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof

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US (2) US11142765B2 (enExample)
EP (1) EP3555294A4 (enExample)
JP (2) JP7233149B2 (enExample)
KR (2) KR102667867B1 (enExample)
CN (2) CN118421624A (enExample)
AU (2) AU2017377661B2 (enExample)
BR (1) BR112019012312A8 (enExample)
CA (1) CA3047154A1 (enExample)
IL (2) IL304674B2 (enExample)
MX (2) MX2019007110A (enExample)
RU (1) RU2763319C2 (enExample)
WO (1) WO2018107228A1 (enExample)
ZA (1) ZA202106827B (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11142765B2 (en) * 2016-12-14 2021-10-12 Benitec Ip Holdings, Inc. Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof
CA3114945A1 (en) * 2018-10-17 2020-04-23 Benitec Biopharma Limited Methods for treating oculopharyngeal muscular dystrophy (opmd)
EP3931317A4 (en) 2019-02-28 2023-01-04 Benitec IP Holdings Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD)
IL294132A (en) * 2019-12-23 2022-08-01 Versameb Ag Compositions and methods for simultaneously modulating expression of genes

Citations (1)

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WO2010105372A1 (en) * 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression

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EP1783645A1 (en) * 2005-11-08 2007-05-09 Actigenics Methods for the identification of microRNA and their applications in research and human health
BRPI0807047A2 (pt) * 2007-01-31 2014-04-22 Basf Plant Science Gmbh Método para o aumento da resistência ao estresse abiótico em plantas relativa as plantas de controle, e, usos de uma construção, e de um ácido nucleico que codifica um polipeptídeo semelhante a nap1.
PT2164967E (pt) * 2007-05-31 2015-10-27 Univ Iowa Res Found Redução da toxicidade não dirigida do arn de interferência
JP6170077B2 (ja) * 2012-02-16 2017-07-26 エータイアー ファーマ， インコーポレイテッド 自己免疫および炎症疾患を処置するためのヒスチジルｔＲＮＡ合成酵素
WO2014077693A1 (en) * 2012-11-16 2014-05-22 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Means and methods for reducing an effect of aging in a mammalian cell
AU2014205036A1 (en) * 2013-01-08 2015-07-30 Benitec Biopharma Limited Age-related macular degeneration treatment
KR102353847B1 (ko) 2016-04-14 2022-01-21 베니텍 바이오파마 리미티드 안구인두 근이영양증(opmd)의 치료용 시약 및 이의 용도
US11142765B2 (en) 2016-12-14 2021-10-12 Benitec Ip Holdings, Inc. Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof

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WO2010105372A1 (en) * 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression

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ANVAR, S.Y. et al., "A decline in PABPN1 induces progressive muscle weakness in Oculopharyngeal muscle dystrophy and in muscle again", 2013. AGING, Vol.5, No.6, pages 412-426. *
APPONI, L.H. et al., "Loss of nuclear poly(A)-binding protein 1 causes defects in myogenesis and mRNA biogenesis", 2010. HUMAN MOLECULAR GENETICS, Vol.19, No.6, pages 1058-1065. *

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BR112019012312A2 (pt) 2020-02-11
AU2017377661A1 (en) 2019-07-18
KR20190104151A (ko) 2019-09-06
JP7233149B2 (ja) 2023-03-06
MX2019007110A (es) 2019-12-05
RU2763319C2 (ru) 2021-12-28
MX2024003643A (es) 2024-04-15
KR102495222B1 (ko) 2023-02-09
CN118421624A (zh) 2024-08-02
US11142765B2 (en) 2021-10-12
IL304674B2 (en) 2024-11-01
IL267353A (en) 2019-08-29
US11932852B2 (en) 2024-03-19
WO2018107228A1 (en) 2018-06-21
CN110382698B (zh) 2024-05-10
AU2023222827A1 (en) 2023-09-14
JP2020503014A (ja) 2020-01-30
IL304674A (en) 2023-09-01
CN110382698A (zh) 2019-10-25
JP2023062092A (ja) 2023-05-02
IL304674B1 (en) 2024-07-01
EP3555294A4 (en) 2020-08-26
CA3047154A1 (en) 2018-06-21
RU2019121950A (ru) 2021-01-18
EP3555294A1 (en) 2019-10-23
AU2023222827B2 (en) 2025-02-27
IL267353B2 (en) 2023-12-01
KR20230020586A (ko) 2023-02-10
IL267353B1 (en) 2023-08-01
ZA202106827B (en) 2022-07-27
KR102667867B1 (ko) 2024-05-29
RU2019121950A3 (enExample) 2021-01-18
JP7431359B2 (ja) 2024-02-14
BR112019012312A8 (pt) 2023-02-28
US20220056446A1 (en) 2022-02-24
US20190330629A1 (en) 2019-10-31

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