AU2020229886B2 - Compositions and methods for treating oculopharyngeal muscular dystrophy (OPMD) - Google Patents

Compositions and methods for treating oculopharyngeal muscular dystrophy (OPMD)

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Publication number
AU2020229886B2
AU2020229886B2 AU2020229886A AU2020229886A AU2020229886B2 AU 2020229886 B2 AU2020229886 B2 AU 2020229886B2 AU 2020229886 A AU2020229886 A AU 2020229886A AU 2020229886 A AU2020229886 A AU 2020229886A AU 2020229886 B2 AU2020229886 B2 AU 2020229886B2
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seq
set forth
sequence set
shmir
sequence
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AU2020229886A1 (en
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Shih-Chu KAO
Petrus W. Roelvink
Vanessa STRINGS-UFOMBAH
David Suhy
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Benitec IP Holdings Inc
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Benitec IP Holdings Inc
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
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    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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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
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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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
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    • C12N2710/14011—Baculoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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AU2020229886A 2019-02-28 2020-02-28 Compositions and methods for treating oculopharyngeal muscular dystrophy (OPMD) Active AU2020229886B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962812187P 2019-02-28 2019-02-28
US62/812,187 2019-02-28
PCT/AU2020/050182 WO2020172720A1 (en) 2019-02-28 2020-02-28 Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd)

Publications (2)

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AU2020229886A1 AU2020229886A1 (en) 2021-09-09
AU2020229886B2 true AU2020229886B2 (en) 2025-09-18

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AU2020229886A Active AU2020229886B2 (en) 2019-02-28 2020-02-28 Compositions and methods for treating oculopharyngeal muscular dystrophy (OPMD)

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US (1) US20220098614A1 (enExample)
EP (1) EP3931317A4 (enExample)
JP (1) JP2022522166A (enExample)
CN (2) CN113631706A (enExample)
AU (1) AU2020229886B2 (enExample)
BR (1) BR112021017054A2 (enExample)
CA (1) CA3130221A1 (enExample)
IL (1) IL285841A (enExample)
MX (1) MX2021010412A (enExample)
SG (1) SG11202108469XA (enExample)
WO (1) WO2020172720A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022508835A (ja) * 2018-10-17 2022-01-19 ベニテック バイオファーマ リミテッド 眼咽頭筋ジストロフィー（ｏｐｍｄ）を治療するための方法
AU2024310011A1 (en) * 2023-06-28 2026-02-05 Benitec IP Holdings Inc. Device and methods for administering a therapeutic composition to the pharyngeal muscle
WO2026011009A1 (en) * 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders

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WO2018107228A1 (en) * 2016-12-14 2018-06-21 Benitec Biopharma Limited Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof

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AU2003212708A1 (en) 2002-03-05 2003-09-16 Stichting Voor De Technische Wetenschappen Baculovirus expression system
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JP7496667B2 (ja) 2016-04-21 2024-06-07 ビロベク，インコーポレイテッド 昆虫細胞中でのａａｖ生成、方法およびその組成物
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Publication number Publication date
JP2022522166A (ja) 2022-04-14
US20220098614A1 (en) 2022-03-31
BR112021017054A2 (pt) 2021-11-09
SG11202108469XA (en) 2021-09-29
IL285841A (en) 2021-10-31
WO2020172720A1 (en) 2020-09-03
CN121160804A (zh) 2025-12-19
EP3931317A4 (en) 2023-01-04
MX2021010412A (es) 2022-01-24
CA3130221A1 (en) 2020-09-03
CN113631706A (zh) 2021-11-09
KR20210138030A (ko) 2021-11-18
EP3931317A1 (en) 2022-01-05
AU2020229886A1 (en) 2021-09-09

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