AU2021358957A1 - Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21 - Google Patents

Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21 Download PDF

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Publication number
AU2021358957A1
AU2021358957A1 AU2021358957A AU2021358957A AU2021358957A1 AU 2021358957 A1 AU2021358957 A1 AU 2021358957A1 AU 2021358957 A AU2021358957 A AU 2021358957A AU 2021358957 A AU2021358957 A AU 2021358957A AU 2021358957 A1 AU2021358957 A1 AU 2021358957A1
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nucleic acid
vector
promoter
seq
fkrp
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AU2021358957A9 (en
Inventor
Martin K. Childers
Sinclair COOPER
Antonia EVRIPIOTI
Juan Manuel IGLESIAS GONZALEZ
SiewHui LOW
Anh Nguyen
Michael L. Roberts
Lester SUAREZ
Anna Tretiakova
Xiao Xiao
Jorge Omar YANEZ-CUNA
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University of North Carolina at Chapel Hill
AskBio Inc
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University of North Carolina at Chapel Hill
Asklepios Biopharmaceutical Inc
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Publication of AU2021358957A9 publication Critical patent/AU2021358957A9/en
Assigned to THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL, AskBio Inc. reassignment THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL Amend patent request/document other than specification (104) Assignors: ASKLEPIOS BIOPHARMACEUTICAL, INC., THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/08—Transferases for other substituted phosphate groups (2.7.8)
    • 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
    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • 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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    • A01K2217/07—Animals genetically altered by homologous recombination
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    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • 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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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Physical Education & Sports Medicine (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2021358957A 2020-10-07 2021-10-06 Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21 Pending AU2021358957A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063088757P 2020-10-07 2020-10-07
US63/088,757 2020-10-07
US202163214123P 2021-06-23 2021-06-23
US63/214,123 2021-06-23
US202163229726P 2021-08-05 2021-08-05
US63/229,726 2021-08-05
PCT/US2021/053768 WO2022076556A2 (en) 2020-10-07 2021-10-06 Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i)

Publications (2)

Publication Number Publication Date
AU2021358957A1 true AU2021358957A1 (en) 2023-06-08
AU2021358957A9 AU2021358957A9 (en) 2024-02-08

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AU2021358957A Pending AU2021358957A1 (en) 2020-10-07 2021-10-06 Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21

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US (1) US20230374542A1 (https=)
EP (1) EP4225382A4 (https=)
JP (1) JP2023545731A (https=)
AU (1) AU2021358957A1 (https=)
CA (1) CA3195177A1 (https=)
IL (1) IL301955A (https=)
WO (1) WO2022076556A2 (https=)

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IL322735A (en) * 2023-02-13 2025-10-01 Astrazeneca Ireland Ltd Nucleic acids encoding BCL2-associated athenogen 3 (BAG3) for gene therapy

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US20230374542A1 (en) 2023-11-23
EP4225382A2 (en) 2023-08-16
IL301955A (en) 2023-06-01
WO2022076556A2 (en) 2022-04-14
CA3195177A1 (en) 2022-04-14
JP2023545731A (ja) 2023-10-31
WO2022076556A3 (en) 2022-05-19
EP4225382A4 (en) 2025-04-23
AU2021358957A9 (en) 2024-02-08

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