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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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
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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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    • 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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    • 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
    • C—CHEMISTRY; METALLURGY
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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
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    • 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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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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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)

Publication Number Publication Date
CA3130221A1 true CA3130221A1 (en) 2020-09-03

Family

ID=72238736

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

Country Status (11)

Country Link
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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US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
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
WO2003042361A2 (en) 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
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
AU2003212708A1 (en) 2002-03-05 2003-09-16 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
US8008468B2 (en) 2005-02-16 2011-08-30 Benitec, Inc. RNAi expression constructs with liver-specific enhancer/promoter
EP2311966A3 (en) 2005-10-20 2012-09-05 Amsterdam Molecular Therapeutics (AMT) B.V. Improved AAV vectors produced in insect cells
WO2007120533A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Minigene expression cassette
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
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ES2751999T3 (es) 2008-01-29 2020-04-02 Applied Genetic Tech Corporation Producción recombinante de virus adeno-asociados usando células BHK en suspensión
EP3024497B1 (en) * 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
WO2015031686A1 (en) 2013-08-30 2015-03-05 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
CN104815338A (zh) * 2015-05-01 2015-08-05 许瑞安 一种携带kal基因重组腺相关病毒载体的基因治疗药物
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
JP7233149B2 (ja) * 2016-12-14 2023-03-06 ベニテック アイピー ホールディングス インコーポレーテッド 眼咽頭型筋ジストロフィー（ｏｐｍｄ）を治療するための試薬及びその使用
EP3575398A4 (en) * 2017-01-30 2020-11-11 Nippon Medical School Foundation ADENO-ASSOCIATED VIRUS (AAV) CAPSIDE PROTEIN MUTANT
BR112020003976A8 (pt) 2017-08-31 2023-03-28 Benitec Biopharma Ltd Vírus adeno-associado (aav) com domínio fosfolipase modificado

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Publication number Publication date
AU2020229886B2 (en) 2025-09-18
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
CN113631706A (zh) 2021-11-09
KR20210138030A (ko) 2021-11-18
EP3931317A1 (en) 2022-01-05
AU2020229886A1 (en) 2021-09-09

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