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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    • C07K—PEPTIDES
    • 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
    • A01K2267/00—Animals characterised by purpose
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    • C07K2319/00—Fusion polypeptide
    • 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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    • C12N2750/14011—Parvoviridae
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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 (https=)
EP (1) EP3931317A4 (https=)
JP (1) JP2022522166A (https=)
CN (2) CN121160804A (https=)
AU (1) AU2020229886B2 (https=)
BR (1) BR112021017054A2 (https=)
CA (1) CA3130221A1 (https=)
IL (1) IL285841A (https=)
MX (1) MX2021010412A (https=)
SG (1) SG11202108469XA (https=)
WO (1) WO2020172720A1 (https=)

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CN112867511A (zh) * 2018-10-17 2021-05-28 贝尼泰克生物制药有限公司 用于治疗眼咽肌营养不良(opmd)的方法
KR20260030147A (ko) * 2023-06-28 2026-03-05 베니텍 아이피 홀딩스 아이엔씨. 인두 근육에 치료 조성물을 투여하기 위한 장치 및 방법
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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Publication number Publication date
CA3130221A1 (en) 2020-09-03
KR20210138030A (ko) 2021-11-18
IL285841A (en) 2021-10-31
CN113631706A (zh) 2021-11-09
EP3931317A4 (en) 2023-01-04
JP2022522166A (ja) 2022-04-14
AU2020229886A1 (en) 2021-09-09
WO2020172720A1 (en) 2020-09-03
US20220098614A1 (en) 2022-03-31
SG11202108469XA (en) 2021-09-29
CN121160804A (zh) 2025-12-19
BR112021017054A2 (pt) 2021-11-09
MX2021010412A (es) 2022-01-24
EP3931317A1 (en) 2022-01-05

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