CA3128876A1 - Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease - Google Patents

Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease Download PDF

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Publication number
CA3128876A1
CA3128876A1 CA3128876A CA3128876A CA3128876A1 CA 3128876 A1 CA3128876 A1 CA 3128876A1 CA 3128876 A CA3128876 A CA 3128876A CA 3128876 A CA3128876 A CA 3128876A CA 3128876 A1 CA3128876 A1 CA 3128876A1
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Canada
Prior art keywords
base editor
gene
adenosine
nucleic acid
snp associated
Prior art date
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Pending
Application number
CA3128876A
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English (en)
French (fr)
Inventor
Ian SLAYMAKER
Nicole GAUDELLI
Yi Yu
Bernd ZETSCHE
David A. BORN
Seung-Joo Lee
Michael Packer
Jason Michael GEHRKE
Natalie PETROSSIAN
Angelica Messana
Shaunna BERKOVITCH
David Bryson
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Beam Therapeutics Inc
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Beam Therapeutics Inc
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Publication of CA3128876A1 publication Critical patent/CA3128876A1/en
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/50—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
    • 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
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    • 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
    • C12N15/1137—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 against enzymes
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
    • C—CHEMISTRY; METALLURGY
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    • C12Y—ENZYMES
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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CA3128876A 2019-02-13 2020-02-13 Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease Pending CA3128876A1 (en)

Applications Claiming Priority (19)

Application Number Priority Date Filing Date Title
US201962805271P 2019-02-13 2019-02-13
US62/805,271 2019-02-13
US201962850919P 2019-05-21 2019-05-21
US62/850,919 2019-05-21
US201962852224P 2019-05-23 2019-05-23
US201962852228P 2019-05-23 2019-05-23
US62/852,228 2019-05-23
US62/852,224 2019-05-23
US201962873138P 2019-07-11 2019-07-11
US62/873,138 2019-07-11
US201962888867P 2019-08-19 2019-08-19
US62/888,867 2019-08-19
US201962931722P 2019-11-06 2019-11-06
US62/931,722 2019-11-06
US201962941569P 2019-11-27 2019-11-27
US62/941,569 2019-11-27
US202062966526P 2020-01-27 2020-01-27
US62/966,526 2020-01-27
PCT/US2020/018073 WO2020168051A1 (en) 2019-02-13 2020-02-13 Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease

Publications (1)

Publication Number Publication Date
CA3128876A1 true CA3128876A1 (en) 2020-08-20

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Application Number Title Priority Date Filing Date
CA3128876A Pending CA3128876A1 (en) 2019-02-13 2020-02-13 Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease

Country Status (8)

Country Link
US (1) US20230140953A1 (https=)
EP (1) EP3924484A4 (https=)
JP (2) JP7586601B2 (https=)
KR (1) KR20210127206A (https=)
CN (2) CN114040970B (https=)
AU (1) AU2020223306A1 (https=)
CA (1) CA3128876A1 (https=)
WO (1) WO2020168051A1 (https=)

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EP3956449B1 (en) 2019-04-15 2026-02-25 EdiGene Therapeutics (Beijing) Inc. Methods and compositions for editing rnas
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KR20210127206A (ko) 2021-10-21
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