AU2019265019B2 - Methods of substituting pathogenic amino acids using programmable base editor systems - Google Patents

Methods of substituting pathogenic amino acids using programmable base editor systems

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Publication number
AU2019265019B2
AU2019265019B2 AU2019265019A AU2019265019A AU2019265019B2 AU 2019265019 B2 AU2019265019 B2 AU 2019265019B2 AU 2019265019 A AU2019265019 A AU 2019265019A AU 2019265019 A AU2019265019 A AU 2019265019A AU 2019265019 B2 AU2019265019 B2 AU 2019265019B2
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Prior art keywords
cell
domain
base
base editor
deaminase
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AU2019265019A
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AU2019265019A1 (en
Inventor
David Bryson
John Evans
Yanfang FU
Nicole GAUDELLI
Jason Michael GEHRKE
J. Keith Joung
Michael Packer
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Beam Therapeutics Inc
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Beam Therapeutics Inc
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Priority to AU2026200834A priority Critical patent/AU2026200834A1/en
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    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
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    • C12N2800/00—Nucleic acids vectors
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AU2019265019A 2018-05-11 2019-05-11 Methods of substituting pathogenic amino acids using programmable base editor systems Active AU2019265019B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2026200834A AU2026200834A1 (en) 2018-05-11 2026-02-05 Methods of substituting pathogenic amino acids using programmable base editor systems

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862670539P 2018-05-11 2018-05-11
US201862670521P 2018-05-11 2018-05-11
US62/670,521 2018-05-11
US62/670,539 2018-05-11
US201862780890P 2018-12-17 2018-12-17
US62/780,890 2018-12-17
PCT/US2019/031897 WO2019217942A1 (en) 2018-05-11 2019-05-11 Methods of substituting pathogenic amino acids using programmable base editor systems

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AU2019265019A1 AU2019265019A1 (en) 2020-11-26
AU2019265019B2 true AU2019265019B2 (en) 2025-11-06

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US (2) US12133884B2 (https=)
EP (1) EP3799603A4 (https=)
JP (2) JP7642531B2 (https=)
KR (2) KR20250134703A (https=)
CN (2) CN121555430A (https=)
AU (2) AU2019265019B2 (https=)
CA (1) CA3100019A1 (https=)
WO (1) WO2019217942A1 (https=)

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US20220401530A1 (en) 2022-12-22
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AU2019265019A1 (en) 2020-11-26
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