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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    • 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
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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)
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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)
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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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  • 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (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 (enExample)
EP (1) EP3972654A4 (enExample)
JP (1) JP7679311B2 (enExample)
KR (1) KR20220010540A (enExample)
CN (2) CN114206395B (enExample)
AU (1) AU2020279751A1 (enExample)
CA (1) CA3140093A1 (enExample)
WO (1) WO2020236936A1 (enExample)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3765615T3 (pl) 2018-03-14 2023-11-13 Arbor Biotechnologies, Inc. Nowe enzymy i układy crispr ukierunkowane na dna
WO2019217942A1 (en) 2018-05-11 2019-11-14 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
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
EP3924479A4 (en) * 2019-02-13 2023-01-25 Beam Therapeutics, Inc. ADENOSINE DEAMINASE BASE EDITORS AND METHODS OF USING THEM TO MODIFY A NUCLEOBASE IN A TARGET SEQUENCE
WO2020168135A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
US12600971B2 (en) 2019-02-13 2026-04-14 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
US12594301B2 (en) 2019-09-27 2026-04-07 Beam Therapeutics Inc. Compositions and methods for treatment of liquid cancers
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
EP4396207A4 (en) * 2021-09-03 2025-07-16 Univ Chicago POLYPEPTIDES AND METHODS FOR MODIFYING NUCLEIC ACIDS
EP4404942A4 (en) * 2021-09-26 2026-06-17 Wave Life Sciences Ltd Compositions for the Editing of Transcripts MECP2 and Related Processes
WO2023140694A1 (ko) * 2022-01-24 2023-07-27 주식회사 툴젠 스트렙토코커스 피오게네스 유래 cas9 변이체
CA3266991A1 (en) * 2022-09-08 2024-03-14 The University Court Of The University Of Edinburgh TREATMENT OF RETT SYNDROME
WO2024102811A2 (en) * 2022-11-08 2024-05-16 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
JP2026524490A (ja) * 2023-07-07 2026-07-22 ミリアド ジェネティクス，インコーポレーテッド 乳がんについての多祖先ポリジェニックリスク評価
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
SG11201510327TA (en) * 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
KR20250012194A (ko) * 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
EP3957731A1 (en) * 2015-07-15 2022-02-23 Rutgers, The State University of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
WO2018027078A1 (en) * 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
RU2019133002A (ru) * 2017-03-24 2021-04-26 Зе Юнивёрсити Корт Оф Зе Юнивёрсити Оф Эдинбург ЭКСПРЕССИОННЫЕ КАССЕТЫ MeCP2
IL316453A (en) * 2017-10-06 2024-12-01 Univ Oregon Health & Science RNA editing agents and methods
CA3082251A1 (en) * 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
EP3699267A4 (en) 2017-10-17 2021-10-27 Kyoto University METHOD OF MANUFACTURING AN ARTIFICIAL NEUROMUSCULAR END PLATE FROM PLURIPOTENT STEM CELLS
KR102853088B1 (ko) * 2018-05-11 2025-09-02 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법

Also Published As

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

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