CA3140093A1 - Methods of editing a single nucleotide polymorphism using programmable base editor systems - Google Patents

Methods of editing a single nucleotide polymorphism using programmable base editor systems Download PDF

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Publication number
CA3140093A1
CA3140093A1 CA3140093A CA3140093A CA3140093A1 CA 3140093 A1 CA3140093 A1 CA 3140093A1 CA 3140093 A CA3140093 A CA 3140093A CA 3140093 A CA3140093 A CA 3140093A CA 3140093 A1 CA3140093 A1 CA 3140093A1
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Prior art keywords
tada
rett
adenosine deaminase
domain
amino acid
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CA3140093A
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English (en)
French (fr)
Inventor
Jason Michael GEHRKE
Natalie PETROSSIAN
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Beam Therapeutics Inc
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Beam Therapeutics Inc
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Publication of CA3140093A1 publication Critical patent/CA3140093A1/en
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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
    • 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
    • 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
    • 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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    • A61P25/00—Drugs for disorders of the nervous system
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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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)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • 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
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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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA3140093A 2019-05-21 2020-05-20 Methods of editing a single nucleotide polymorphism using programmable base editor systems Pending CA3140093A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962850919P 2019-05-21 2019-05-21
US62/850,919 2019-05-21
PCT/US2020/033807 WO2020236936A1 (en) 2019-05-21 2020-05-20 Methods of editing a single nucleotide polymorphism using programmable base editor systems

Publications (1)

Publication Number Publication Date
CA3140093A1 true CA3140093A1 (en) 2020-11-26

Family

ID=73459015

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3140093A Pending CA3140093A1 (en) 2019-05-21 2020-05-20 Methods of editing a single nucleotide polymorphism using programmable base editor systems

Country Status (8)

Country Link
US (1) US20220387622A1 (https=)
EP (1) EP3972654A4 (https=)
JP (1) JP7679311B2 (https=)
KR (1) KR20220010540A (https=)
CN (2) CN120346348A (https=)
AU (1) AU2020279751A1 (https=)
CA (1) CA3140093A1 (https=)
WO (1) WO2020236936A1 (https=)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019178427A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
WO2019222555A1 (en) 2018-05-16 2019-11-21 Arbor Biotechnologies, Inc. Novel crispr-associated systems and components
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
JP7693552B2 (ja) * 2019-02-13 2025-06-17 ビーム セラピューティクス インク． アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法
CN120174005A (zh) 2019-02-13 2025-06-20 比姆医疗股份有限公司 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
US20240352439A1 (en) * 2021-09-03 2024-10-24 The University Of Chicago Polypeptides and methods for modifying nucleic acids
EP4404942A2 (en) * 2021-09-26 2024-07-31 Wave Life Sciences Ltd. Compositions for editing mecp2 transcripts and methods thereof
US20250154479A1 (en) * 2022-01-24 2025-05-15 Toolgen Incorporated Streptococcus pyogenes-derived cas9 variant
JP2025530183A (ja) * 2022-09-08 2025-09-11 ザ ユニヴァーシティ コート オブ ザ ユニヴァーシティ オブ エディンバラ レット症候群治療法
EP4615574A2 (en) * 2022-11-08 2025-09-17 The Board Of Trustees Of The Leland Stanford Junior University Muscle-specific base editors for correction of mutations causing dilated cardiomyopathy
CN117965505A (zh) * 2023-06-28 2024-05-03 微光基因(苏州)有限公司 工程化的腺苷脱氨酶及碱基编辑器
WO2025014806A1 (en) * 2023-07-07 2025-01-16 Myriad Genetics, Inc. Multiple-ancestry polygenic risk assessment for breast cancer
WO2025199339A2 (en) * 2024-03-20 2025-09-25 Shape Therapeutics Inc. Mecp2-targeting engineered guide rnas and polynucleotides

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX374532B (es) * 2013-06-17 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
CN105683379A (zh) * 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
JP7044373B2 (ja) * 2015-07-15 2022-03-30 ラトガース，ザ ステート ユニバーシティ オブ ニュージャージー ヌクレアーゼ非依存的な標的化遺伝子編集プラットフォームおよびその用途
CN110214183A (zh) * 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
BR112019019833A2 (pt) * 2017-03-24 2020-04-22 The University Court Of The University Of Edinburgh terapia baseada em mecp2
WO2019071274A1 (en) * 2017-10-06 2019-04-11 Oregon Health & Science University Compositions and methods for editing rna
KR20250107288A (ko) * 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
EP3699267A4 (en) 2017-10-17 2021-10-27 Kyoto University PROCESS FOR OBTAINING AN ARTIFICIAL NEUROMUSCULAR JUNCTION FROM PLURIPOTENT STEM CELLS
KR20250133482A (ko) * 2018-05-11 2025-09-05 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법

Also Published As

Publication number Publication date
JP7679311B2 (ja) 2025-05-19
JP2022533673A (ja) 2022-07-25
CN114206395B (zh) 2025-03-28
EP3972654A1 (en) 2022-03-30
US20220387622A1 (en) 2022-12-08
AU2020279751A1 (en) 2021-12-02
WO2020236936A1 (en) 2020-11-26
KR20220010540A (ko) 2022-01-25
EP3972654A4 (en) 2024-07-03
CN114206395A (zh) 2022-03-18
CN120346348A (zh) 2025-07-22

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