CA3090226A1 - Adeno-associated virus compositions for restoring pah gene function and methods of use thereof - Google Patents

Adeno-associated virus compositions for restoring pah gene function and methods of use thereof Download PDF

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Publication number
CA3090226A1
CA3090226A1 CA3090226A CA3090226A CA3090226A1 CA 3090226 A1 CA3090226 A1 CA 3090226A1 CA 3090226 A CA3090226 A CA 3090226A CA 3090226 A CA3090226 A CA 3090226A CA 3090226 A1 CA3090226 A1 CA 3090226A1
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Prior art keywords
amino acid
seq
capsid protein
protein corresponding
nucleotide
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CA3090226A
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English (en)
French (fr)
Inventor
Albert Barnes Seymour
Seemin Seher Ahmed
Jason Boke WRIGHT
Serena Nicole DOLLIVE
James Anthony Mcswiggen
Jaime Michelle Prout
Danielle Lauren Sookiasian
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Homology Medicines Inc
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Homology Medicines Inc
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • A—HUMAN NECESSITIES
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    • C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A—HUMAN NECESSITIES
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/10—Mammal
    • A01K2227/105—Murine
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/00—Animals characterised by purpose
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    • C12N2710/00011—Details
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    • C12N2710/10033—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
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    • C12N2710/10011—Adenoviridae
    • C12N2710/10051—Methods of production or purification of viral material
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • C12N2750/00011—Details
    • 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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  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
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CA3090226A 2018-02-01 2019-02-01 Adeno-associated virus compositions for restoring pah gene function and methods of use thereof Pending CA3090226A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862625149P 2018-02-01 2018-02-01
US62/625,149 2018-02-01
US201862672377P 2018-05-16 2018-05-16
US62/672,377 2018-05-16
PCT/US2019/016354 WO2019152843A1 (en) 2018-02-01 2019-02-01 Adeno-associated virus compositions for restoring pah gene function and methods of use thereof

Publications (1)

Publication Number Publication Date
CA3090226A1 true CA3090226A1 (en) 2019-08-08

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CA3090226A Pending CA3090226A1 (en) 2018-02-01 2019-02-01 Adeno-associated virus compositions for restoring pah gene function and methods of use thereof

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US (3) US20190256867A1 (enExample)
EP (1) EP3746101A4 (enExample)
JP (1) JP2021512871A (enExample)
KR (1) KR20200127170A (enExample)
CN (1) CN111902164A (enExample)
AU (1) AU2019215152A1 (enExample)
BR (1) BR112020015798A2 (enExample)
CA (1) CA3090226A1 (enExample)
CL (1) CL2020002008A1 (enExample)
CO (1) CO2020010793A2 (enExample)
IL (1) IL276368A (enExample)
MX (1) MX2020008133A (enExample)
PE (1) PE20212076A1 (enExample)
WO (1) WO2019152843A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202140791A (zh) * 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
WO2021144649A2 (en) * 2020-01-16 2021-07-22 Takeda Pharmaceutical Company Limited Adeno associated virus based gene therapy for phenylketonuria
WO2021207077A1 (en) * 2020-04-06 2021-10-14 Homology Medicines, Inc. Adeno-associated virus compositions for ids gene transfer and methods of use thereof
TW202208632A (zh) * 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
KR20230079172A (ko) 2020-10-01 2023-06-05 젠자임 코포레이션 간-유도 유전자 대체 요법에 의한 pku의 치료를 위한 인간 pah 발현 카세트

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