JP7646552B2 - 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ - Google Patents

低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ Download PDF

Info

Publication number
JP7646552B2
JP7646552B2 JP2021544294A JP2021544294A JP7646552B2 JP 7646552 B2 JP7646552 B2 JP 7646552B2 JP 2021544294 A JP2021544294 A JP 2021544294A JP 2021544294 A JP2021544294 A JP 2021544294A JP 7646552 B2 JP7646552 B2 JP 7646552B2
Authority
JP
Japan
Prior art keywords
deaminase
cas9
amino acid
fusion protein
domain
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
JP2021544294A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022519507A5 (https=
JP2022519507A (ja
Inventor
イアン スレイメイカー、
ジェイソン マイケル ゲールケ、
ニコル ゴーデッリ、
イ ユ、
Original Assignee
ビーム セラピューティクス インク.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ビーム セラピューティクス インク. filed Critical ビーム セラピューティクス インク.
Publication of JP2022519507A publication Critical patent/JP2022519507A/ja
Publication of JP2022519507A5 publication Critical patent/JP2022519507A5/ja
Priority to JP2025034660A priority Critical patent/JP2025102784A/ja
Application granted granted Critical
Publication of JP7646552B2 publication Critical patent/JP7646552B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C12N9/80Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
    • CCHEMISTRY; METALLURGY
    • C40COMBINATORIAL TECHNOLOGY
    • C40BCOMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00Libraries per se, e.g. arrays, mixtures
    • C40B40/04Libraries containing only organic compounds
    • C40B40/10Libraries containing peptides or polypeptides, or derivatives thereof
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005Cytidine deaminase (3.5.4.5)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021544294A 2019-01-31 2020-01-31 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ Active JP7646552B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025034660A JP2025102784A (ja) 2019-01-31 2025-03-05 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962799702P 2019-01-31 2019-01-31
US62/799,702 2019-01-31
PCT/US2020/016285 WO2020160514A1 (en) 2019-01-31 2020-01-31 Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025034660A Division JP2025102784A (ja) 2019-01-31 2025-03-05 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ

Publications (3)

Publication Number Publication Date
JP2022519507A JP2022519507A (ja) 2022-03-24
JP2022519507A5 JP2022519507A5 (https=) 2023-02-06
JP7646552B2 true JP7646552B2 (ja) 2025-03-17

Family

ID=71842366

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021544294A Active JP7646552B2 (ja) 2019-01-31 2020-01-31 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ
JP2025034660A Pending JP2025102784A (ja) 2019-01-31 2025-03-05 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025034660A Pending JP2025102784A (ja) 2019-01-31 2025-03-05 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ

Country Status (8)

Country Link
US (1) US12612618B2 (https=)
EP (1) EP3918078A4 (https=)
JP (2) JP7646552B2 (https=)
KR (1) KR20210121113A (https=)
CN (1) CN114040977B (https=)
AU (1) AU2020215730A1 (https=)
CA (1) CA3127493A1 (https=)
WO (1) WO2020160514A1 (https=)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
AU2020215730A1 (en) 2019-01-31 2021-07-29 Beam Therapeutics Inc. Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors
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
CN116209756A (zh) 2020-03-04 2023-06-02 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
EP4143315A1 (en) 2020-04-28 2023-03-08 The Broad Institute Inc. <smallcaps/>? ? ?ush2a? ? ? ? ?targeted base editing of thegene
CN116134141A (zh) * 2020-09-04 2023-05-16 国立大学法人神户大学 包含小型化胞苷脱氨酶的双链dna修饰用复合体
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
WO2022120520A1 (en) * 2020-12-07 2022-06-16 Institute Of Zoology, Chinese Academy Of Sciences Engineered cas effector proteins and methods of use thereof
CN113308451B (zh) * 2020-12-07 2023-07-25 中国科学院动物研究所 工程化的Cas效应蛋白及其使用方法
EP4313118A4 (en) * 2021-03-26 2025-06-18 Beam Therapeutics Inc. ADENOSINE DEAMINASE VARIANTS AND THEIR USES
KR20240007651A (ko) * 2021-04-16 2024-01-16 빔 테라퓨틱스, 인크. 간세포의 유전적 변형
WO2023283092A1 (en) * 2021-07-06 2023-01-12 Prime Medicine, Inc. Compositions and methods for efficient genome editing
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
CN114634923B (zh) * 2022-04-07 2024-02-23 尧唐(上海)生物科技有限公司 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途
US20250361530A1 (en) * 2022-05-14 2025-11-27 The Regents Of The University Of California Genome editing systems for multiplexing point mutation introduction in living cells
WO2025119306A1 (zh) * 2023-12-06 2025-06-12 北京齐禾生科生物科技有限公司 优化工程化t细胞的碱基编辑系统及其应用

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017048969A1 (en) 2015-09-17 2017-03-23 The Regents Of The University Of California Variant cas9 polypeptides comprising internal insertions
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018213726A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2019005884A1 (en) 2017-06-26 2019-01-03 The Broad Institute, Inc. CRISPR / CAS-ADENINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED NUCLEIC ACID EDITION

Family Cites Families (140)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9600384D0 (en) 1996-01-09 1996-03-13 Nyfotek As Dna glycosylases
US6886207B1 (en) 1999-06-14 2005-05-03 The Procter & Gamble Company Toothbrush
EP1235914A2 (en) 1999-11-24 2002-09-04 Joseph Rosenecker Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells
US20040003420A1 (en) 2000-11-10 2004-01-01 Ralf Kuhn Modified recombinase
US20050222030A1 (en) 2001-02-21 2005-10-06 Anthony Allison Modified annexin proteins and methods for preventing thrombosis
WO2002068676A2 (en) 2001-02-27 2002-09-06 University Of Rochester METHODS AND COMPOSITIONS FOR MODIFYING APOLIPOPROTEIN B mRNA EDITING
AU2002330714A1 (en) 2001-05-30 2003-01-02 Biomedical Center In silico screening for phenotype-associated expressed sequences
US7300829B2 (en) 2003-06-02 2007-11-27 Applied Materials, Inc. Low temperature process for TFT fabrication
WO2010132092A2 (en) 2009-05-12 2010-11-18 The Scripps Research Institute Cytidine deaminase fusions and related methods
US20110104787A1 (en) 2009-11-05 2011-05-05 President And Fellows Of Harvard College Fusion Peptides That Bind to and Modify Target Nucleic Acid Sequences
EP2513296A4 (en) 2009-12-18 2013-05-22 Univ Leland Stanford Junior USE OF CYTIDINE DAMINASE ASSOCIATED ACTIVITIES TO PROMOTE DEMETHYLATION AND CELL REPROGRAMMEIRING
KR102096534B1 (ko) 2011-09-28 2020-04-03 에라 바이오테크, 에스.에이. 분할된 인테인 및 그의 이용
CN103088008B (zh) 2011-10-31 2014-08-20 中国科学院微生物研究所 胞苷脱氨酶及其编码基因和它们的应用
US20150017136A1 (en) 2013-07-15 2015-01-15 Cellectis Methods for engineering allogeneic and highly active t cell for immunotherapy
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
WO2013188037A2 (en) 2012-06-11 2013-12-19 Agilent Technologies, Inc Method of adaptor-dimer subtraction using a crispr cas6 protein
EP3431497B1 (en) 2012-06-27 2022-07-27 The Trustees of Princeton University Split inteins, conjugates and uses thereof
PL3360964T3 (pl) 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
FI2997141T3 (fi) 2013-05-13 2022-12-15 CD19-spesifinen kimeerinen antigeenireseptori ja sen käyttöjä
WO2014186686A2 (en) 2013-05-17 2014-11-20 Two Blades Foundation Targeted mutagenesis and genome engineering in plants using rna-guided cas nucleases
US9267135B2 (en) 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
EP3666892A1 (en) 2013-07-10 2020-06-17 President and Fellows of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
WO2015021426A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in genome editing
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3835419A1 (en) 2013-12-12 2021-06-16 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
JP6793547B2 (ja) * 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能CRISPR−Cas系による配列操作のための系、方法および組成物
ES2918501T3 (es) 2013-12-19 2022-07-18 Novartis Ag Receptores de antígenos quiméricos de mesotelina humana y usos de los mismos
WO2015092024A2 (en) 2013-12-20 2015-06-25 Cellectis Method of engineering multi-input signal sensitive t cell for immunotherapy
EP3115457B1 (en) 2014-03-05 2019-10-02 National University Corporation Kobe University Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
US20170335281A1 (en) 2014-03-15 2017-11-23 Novartis Ag Treatment of cancer using chimeric antigen receptor
KR20170032406A (ko) 2014-07-15 2017-03-22 주노 쎄러퓨티크스 인코퍼레이티드 입양 세포 치료를 위한 조작된 세포
US10544201B2 (en) 2014-07-31 2020-01-28 Cellectis ROR1 specific multi-chain chimeric antigen receptor
EP3186375A4 (en) 2014-08-28 2019-03-13 North Carolina State University Novel CAS9 PROTEINS AND CHARACTERISTICS FOR DNA TARGETING AND GENOME EDITING
BR112017007765B1 (pt) 2014-10-14 2023-10-03 Halozyme, Inc Composições de adenosina deaminase-2 (ada2), variantes do mesmo e métodos de usar o mesmo
WO2016061368A1 (en) 2014-10-15 2016-04-21 The Children's Hospital Of Philadelphia Compositions and methods for treating b-lymphoid malignancies
US10258697B2 (en) 2014-10-29 2019-04-16 Massachusetts Eye And Ear Infirmary Efficient delivery of therapeutic molecules in vitro and in vivo
PT3216867T (pt) 2014-11-04 2020-07-16 Univ Kobe Nat Univ Corp Método para modificar a sequência de genoma para introduzir mutação específica a sequência de adn alvo por reação de remoção de bases, e complexo molecular nele utilizado
MA40921A (fr) 2014-11-05 2017-09-12 Abbvie Stemcentrx Llc Récepteurs antigéniques chimériques anti-cldn et procédés d'utilisation
WO2016075612A1 (en) 2014-11-12 2016-05-19 Rinat Neuroscience Corp. Inhibitory chimeric antigen receptors
HRP20191873T1 (hr) 2014-12-12 2020-01-24 Bluebird Bio, Inc. Kimerni antigenski receptori
WO2016100955A2 (en) 2014-12-20 2016-06-23 Identifygenomics, Llc Compositions and methods for targeted depletion, enrichment, and partitioning of nucleic acids using crispr/cas system proteins
EP3250689B1 (en) * 2015-01-28 2020-11-04 The Regents of The University of California Methods and compositions for labeling a single-stranded target nucleic acid
MA41613A (fr) 2015-02-23 2018-01-02 Abbvie Stemcentrx Llc Récepteurs antigéniques chimériques anti-dll3 et procédés d'utilisation desdits récepteurs
JP2018522249A (ja) 2015-04-24 2018-08-09 エディタス・メディシン、インコーポレイテッド Cas9分子/ガイドrna分子複合体の評価
US20180291372A1 (en) 2015-05-14 2018-10-11 Massachusetts Institute Of Technology Self-targeting genome editing system
WO2016196388A1 (en) 2015-05-29 2016-12-08 Juno Therapeutics, Inc. Composition and methods for regulating inhibitory interactions in genetically engineered cells
EP3331582A4 (en) 2015-05-29 2019-08-07 Agenovir Corporation METHODS AND COMPOSITIONS FOR THE TREATMENT OF TRANSPLANTATION CELLS
WO2016196655A1 (en) * 2015-06-03 2016-12-08 The Regents Of The University Of California Cas9 variants and methods of use thereof
TWI906646B (zh) * 2015-06-18 2025-12-01 美商博得學院股份有限公司 降低脫靶效應的crispr酶突變
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
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
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
AU2016323985B2 (en) 2015-09-17 2022-12-15 Novartis Ag CAR T cell therapies with enhanced efficacy
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
BR112018008442A2 (pt) 2015-11-05 2018-11-06 Juno Therapeutics Inc receptores quiméricos contendo domínios induto-res de traf e composições e métodos relacionados
US11020429B2 (en) 2015-11-05 2021-06-01 Juno Therapeutics, Inc. Vectors and genetically engineered immune cells expressing metabolic pathway modulators and uses in adoptive cell therapy
PT3408292T (pt) 2016-01-29 2023-07-19 Univ Princeton Inteínas divididas com excecional atividade de splicing
EP3443002A4 (en) 2016-04-14 2019-12-04 Bluebird Bio, Inc. SYSTEMS FOR RECOVERED CHIMERIC ANTIGEN RECEPTORS
KR102546839B1 (ko) 2016-08-03 2023-06-23 워싱턴 유니버시티 키메라 항원 수용체를 이용한 t 세포 악성종양의 치료를 위한 car-t 세포의 유전자 편집
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
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
SG10201913505WA (en) * 2016-10-17 2020-02-27 Univ Nanyang Tech Truncated crispr-cas proteins for dna targeting
WO2018085690A1 (en) 2016-11-04 2018-05-11 Bluebird Bio, Inc. Anti-bcma car t cell compositions
EP3538561A4 (en) 2016-11-11 2020-10-21 The Regents of The University of California RNA-GUIDED VARIANT POLYPEPTIDES AND THEIR METHODS OF USE
US11192929B2 (en) * 2016-12-08 2021-12-07 Regents Of The University Of Minnesota Site-specific DNA base editing using modified APOBEC enzymes
SG10202106058WA (en) 2016-12-08 2021-07-29 Intellia Therapeutics Inc Modified guide rnas
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
KR20230125856A (ko) 2016-12-23 2023-08-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Pcsk9의 유전자 편집
EP3565608A4 (en) 2017-01-05 2020-12-16 Rutgers, The State University of New Jersey TARGETED GENE EDITING PLATFORM INDEPENDENT OF BICATENARY DNA BREAKAGE AND ITS USES
WO2018152197A1 (en) 2017-02-15 2018-08-23 Massachusetts Institute Of Technology Dna writers, molecular recorders and uses thereof
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
US11591589B2 (en) 2017-04-21 2023-02-28 The General Hospital Corporation Variants of Cpf1 (Cas12a) with altered PAM specificity
EP3630849A4 (en) 2017-05-25 2021-01-13 The General Hospital Corporation BIPARTITE BASIC EDITOR (BBE) AND TYPE II-C-CAS9 ZINC FINGER EDITOR ARCHITECTURES
WO2019005886A1 (en) 2017-06-26 2019-01-03 The Broad Institute, Inc. CRISPR / CAS-CYTIDINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED EDITING OF NUCLEIC ACIDS
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
CA3073448A1 (en) 2017-08-23 2019-02-28 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CN109957569B (zh) 2017-12-22 2022-10-25 苏州齐禾生科生物科技有限公司 基于cpf1蛋白的碱基编辑系统和方法
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CA3108281A1 (en) 2018-08-03 2020-02-06 Beam Therapeutics Inc. Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence
EP3841203A4 (en) 2018-08-23 2022-11-02 The Broad Institute Inc. CAS9 VARIANTS WITH NON-CANONICAL PAM SPECIFICITIES AND THEIR USES
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
EP3887521A1 (en) 2018-11-29 2021-10-06 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
AU2020215730A1 (en) 2019-01-31 2021-07-29 Beam Therapeutics Inc. Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors
WO2020160517A1 (en) 2019-01-31 2020-08-06 Beam Therapeutics Inc. Nucleobase editors having reduced off-target deamination and methods of using same to modify a nucleobase target sequence
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
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
US20220098593A1 (en) 2019-02-13 2022-03-31 Beam Therapeutics Inc. Splice acceptor site disruption of a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
WO2020168088A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
CA3128755C (en) 2019-02-13 2024-06-04 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
US20230140953A1 (en) 2019-02-13 2023-05-11 Beam Therapeutics Inc. Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
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
KR20210126680A (ko) 2019-02-13 2021-10-20 빔 테라퓨틱스, 인크. 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
KR102258713B1 (ko) 2019-07-31 2021-05-31 한양대학교 산학협력단 사이토신 염기교정용 조성물 및 이의 용도
AU2020344547A1 (en) 2019-09-09 2022-03-24 Beam Therapeutics Inc. Novel nucleobase editors and methods of using same
AU2020466994A1 (en) 2019-09-17 2022-04-21 Rutgers, The State University Of New Jersey Highly efficient DNA base editors mediated by RNA-aptamer recruitment for targeted genome modification and uses thereof
EP4051788A4 (en) 2019-10-30 2023-12-06 Pairwise Plants Services, Inc. TYPE V CRISPR CAS BASE EDITORS AND METHODS OF USE THEREOF
US20230086199A1 (en) 2019-11-26 2023-03-23 The Broad Institute, Inc. Systems and methods for evaluating cas9-independent off-target editing of nucleic acids
EP4100032B1 (en) 2020-02-05 2025-10-15 The Broad Institute, Inc. Gene editing methods for treating spinal muscular atrophy
EP4100519A2 (en) 2020-02-05 2022-12-14 The Broad Institute, Inc. Adenine base editors and uses thereof
JP2023517890A (ja) 2020-03-04 2023-04-27 スージョウ チー バイオデザイン バイオテクノロジー カンパニー リミテッド 改善されたシトシン塩基編集システム
JP2023518395A (ja) 2020-03-19 2023-05-01 インテリア セラピューティクス,インコーポレイテッド 指向性ゲノム編集のための方法及び組成物
EP4132951A4 (en) 2020-04-09 2025-06-11 Verve Therapeutics, Inc. CHEMICALLY MODIFIED GENE EDITING GUIDE RNAS WITH CAS12B
WO2022008935A1 (en) 2020-07-10 2022-01-13 Horizon Discovery Limited Method for producing genetically modified cells
US20240247242A1 (en) 2020-07-15 2024-07-25 LifeEDIT Therapeutics, Inc. Uracil stabilizing proteins and active fragments and variants thereof and methods of use
JP2023540797A (ja) 2020-09-11 2023-09-26 メタゲノミ,インコーポレイテッド 塩基編集酵素
MX2023002848A (es) 2020-09-11 2023-03-22 Lifeedit Therapeutics Inc Enzimas modificadoras del acido desoxirribonucleico (adn) y fragmentos activos y sus variantes y metodos de uso.
IT202000028688A1 (it) 2020-11-27 2022-05-27 Consiglio Nazionale Ricerche Varianti della citidina deaminasi per l’editazione di basi
CA3207144A1 (en) 2021-01-05 2022-07-14 Horizon Discovery Limited Method for producing genetically modified cells
JP2024502348A (ja) 2021-01-05 2024-01-18 ホライズン・ディスカバリー・リミテッド Tracr不含 V型Casシステム用のガイドRNAデザインおよび複合体
WO2022150372A1 (en) 2021-01-05 2022-07-14 Horizon Discovery Ltd. Guide rna designs and complexes for type v cas systems
CA3208612A1 (en) 2021-02-19 2022-08-25 Beam Therapeutics Inc. Recombinant rabies viruses for gene therapy
EP4313118A4 (en) 2021-03-26 2025-06-18 Beam Therapeutics Inc. ADENOSINE DEAMINASE VARIANTS AND THEIR USES
US20240327859A1 (en) 2021-07-02 2024-10-03 University Of Maryland, College Park Cytidine deaminases and methods of genome editing using the same
CA3225808A1 (en) 2021-07-16 2023-01-19 David R. Liu Context-specific adenine base editors and uses thereof
US20240352439A1 (en) 2021-09-03 2024-10-24 The University Of Chicago Polypeptides and methods for modifying nucleic acids
IL311607A (en) 2021-09-22 2024-05-01 Verve Therapeutics Inc PCSK9 or ANGPTL3 gene editing and preparations and methods for using them to treat the disease
US20230105319A1 (en) 2021-09-24 2023-04-06 Crispr Therapeutics Ag PRODRUG INCORPORATED sgRNA SYNTHESIS
WO2023125814A1 (zh) 2021-12-29 2023-07-06 华东师范大学 腺嘌呤脱氨酶及其应用
CN119013310A (zh) 2022-02-17 2024-11-22 正序(上海)生物科技有限公司 具有改善的编辑精确度的突变的胞嘧啶脱氨酶
CN114634923B (zh) 2022-04-07 2024-02-23 尧唐(上海)生物科技有限公司 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途
EP4532704A2 (en) 2022-05-26 2025-04-09 UCB Biopharma SRL Novel nucleic acid-editing proteins
EP4540378A1 (en) 2022-06-20 2025-04-23 CRISPR Therapeutics AG Base editing proteins and uses thereof
AU2023288909A1 (en) 2022-06-23 2025-01-09 Basf Agricultural Solutions Us Llc Diversifying base editing
WO2024040083A1 (en) 2022-08-16 2024-02-22 The Broad Institute, Inc. Evolved cytosine deaminases and methods of editing dna using same
KR20250075667A (ko) 2022-09-23 2025-05-28 기초과학연구원 신규한 아데닌 탈아미노효소 변이체 및 이를 사용한 염기 교정 방법
EP4594486A2 (en) 2022-09-26 2025-08-06 Beam Therapeutics Inc. Synthetic polypeptides and uses thereof
WO2024179426A2 (en) 2023-02-28 2024-09-06 Accuredit Therapeutics (Suzhou) Co., Ltd. Deaminases for use in base editing
EP4688814A1 (en) 2023-04-27 2026-02-11 University of Massachusetts Cas-embedded cytidine deaminase ribonucleoprotein complexes having improved base editing specificity and efficiency
CN121079412A (zh) 2023-04-28 2025-12-05 比姆医疗股份有限公司 经修饰的指导rna
AU2024303631A1 (en) 2023-06-15 2025-12-11 Beam Therapeutics Inc. Methods and compositions for treating alpha-1 antitrypsin deficiency
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

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017048969A1 (en) 2015-09-17 2017-03-23 The Regents Of The University Of California Variant cas9 polypeptides comprising internal insertions
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018213726A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2019005884A1 (en) 2017-06-26 2019-01-03 The Broad Institute, Inc. CRISPR / CAS-ADENINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED NUCLEIC ACID EDITION

Also Published As

Publication number Publication date
CN114040977A (zh) 2022-02-11
US20220098572A1 (en) 2022-03-31
EP3918078A1 (en) 2021-12-08
JP2025102784A (ja) 2025-07-08
CN114040977B (zh) 2024-09-03
US12612618B2 (en) 2026-04-28
EP3918078A4 (en) 2022-12-14
KR20210121113A (ko) 2021-10-07
JP2022519507A (ja) 2022-03-24
AU2020215730A1 (en) 2021-07-29
WO2020160514A1 (en) 2020-08-06
CA3127493A1 (en) 2020-08-06

Similar Documents

Publication Publication Date Title
JP7646552B2 (ja) 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ
JP7657711B2 (ja) 核酸塩基編集システムを送達するための組成物および方法
JP7717684B2 (ja) 新規核酸塩基エディター及びその使用方法
JP7679311B2 (ja) プログラム可能塩基エディターシステムを用いた一塩基多型編集法
JP7646554B2 (ja) アルファ-1アンチトリプシン不全を治療するための組成物および方法
CN114026237B (zh) 用于治疗1a型糖原贮积病的组成物和方法
EP3924481A1 (en) Compositions and methods for treating hemoglobinopathies
CN114072509A (zh) 脱氨反应脱靶减低的核碱基编辑器和使用其修饰核碱基靶序列的方法
KR20230158476A (ko) 유전자 요법을 위한 재조합 광견병 바이러스
CN114929287A (zh) 用于治疗乙型肝炎的组合物和方法
AU2021360919A1 (en) Compositions and methods for treating glycogen storage disease type 1a
JP2025533556A (ja) 合成ポリペプチド及びその用途

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230127

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230127

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20231220

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240321

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240702

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241001

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20250204

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250305

R150 Certificate of patent or registration of utility model

Ref document number: 7646552

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150