CA3130221A1 - Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd) - Google Patents

Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd) Download PDF

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Publication number
CA3130221A1
CA3130221A1 CA3130221A CA3130221A CA3130221A1 CA 3130221 A1 CA3130221 A1 CA 3130221A1 CA 3130221 A CA3130221 A CA 3130221A CA 3130221 A CA3130221 A CA 3130221A CA 3130221 A1 CA3130221 A1 CA 3130221A1
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Canada
Prior art keywords
seq
set forth
sequence set
sequence
shmir
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CA3130221A
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English (en)
French (fr)
Inventor
Vanessa STRINGS-UFOMBAH
David Suhy
Shih-Chu KAO
Petrus W. Roelvink
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Benitec IP Holdings Inc
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Benitec IP Holdings Inc
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Publication of CA3130221A1 publication Critical patent/CA3130221A1/en
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • 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
    • 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
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
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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
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    • C12N2710/14011—Baculoviridae
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA3130221A 2019-02-28 2020-02-28 Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd) Pending CA3130221A1 (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 (1)

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CA3130221A1 true CA3130221A1 (en) 2020-09-03

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CA3130221A Pending CA3130221A1 (en) 2019-02-28 2020-02-28 Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd)

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Country Link
US (1) US20220098614A1 (enExample)
EP (1) EP3931317A4 (enExample)
JP (1) JP7873074B2 (enExample)
KR (1) KR102959609B1 (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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3866859A4 (en) * 2018-10-17 2023-08-09 Benitec IP Holdings Inc. METHODS OF TREATMENT FOR OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD)
EP4493703A4 (en) * 2022-03-18 2026-08-05 Univ Florida METHODS AND COMPOSITIONS FOR TREATING TMEM43-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR
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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JPH11514853A (ja) 1995-09-08 1999-12-21 ジエンザイム コーポレイション 遺伝子治療のための改良されたａａｖベクター
US7208315B2 (en) 2000-01-05 2007-04-24 Fred Hutchinson Cancer Research Center Compositions and methods for efficient AAV vector production
EP1390490B1 (en) 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
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WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
US20060166363A1 (en) * 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
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WO2007120533A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Minigene expression cassette
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WO2007148971A2 (en) 2006-06-21 2007-12-27 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
EP2242840B1 (en) 2008-01-29 2019-07-24 Applied Genetic Technologies Corporation Recombinant adeno-associated virus production using bhk cells in suspension
RU2664471C2 (ru) * 2013-07-26 2018-08-17 Юниверсити Оф Айова Рисерч Фаундейшн Способы и композиции для лечения болезней мозга
US11078464B2 (en) 2013-08-30 2021-08-03 Amgen Inc. High titer recombinant AAV vector production in adherent and suspension cells
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EP3575398A4 (en) 2017-01-30 2020-11-11 Nippon Medical School Foundation ADENO-ASSOCIATED VIRUS (AAV) CAPSIDE PROTEIN MUTANT
UY37863A (es) 2017-08-31 2019-03-29 Benitec Biopharma Ltd Virus adenoasociado (aav) con dominio de fosfolipasa modificado

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Publication number Publication date
SG11202108469XA (en) 2021-09-29
JP7873074B2 (ja) 2026-06-11
AU2020229886B2 (en) 2025-09-18
JP2022522166A (ja) 2022-04-14
CN121160804A (zh) 2025-12-19
BR112021017054A2 (pt) 2021-11-09
KR20210138030A (ko) 2021-11-18
CN113631706A (zh) 2021-11-09
IL285841A (en) 2021-10-31
EP3931317A4 (en) 2023-01-04
MX2021010412A (es) 2022-01-24
KR102959609B1 (ko) 2026-04-29
EP3931317A1 (en) 2022-01-05
US20220098614A1 (en) 2022-03-31
AU2020229886A1 (en) 2021-09-09
WO2020172720A1 (en) 2020-09-03

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