CA3112011A1 - Compositions and methods for delivering a nucleobase editing system - Google Patents

Compositions and methods for delivering a nucleobase editing system Download PDF

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Publication number
CA3112011A1
CA3112011A1 CA3112011A CA3112011A CA3112011A1 CA 3112011 A1 CA3112011 A1 CA 3112011A1 CA 3112011 A CA3112011 A CA 3112011A CA 3112011 A CA3112011 A CA 3112011A CA 3112011 A1 CA3112011 A1 CA 3112011A1
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Prior art keywords
cas9
terminal fragment
fragment
deaminase
fused
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CA3112011A
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English (en)
French (fr)
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David Bryson
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Beam Therapeutics Inc
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Beam Therapeutics Inc
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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
    • C12N15/62—DNA sequences coding for fusion proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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]
    • C—CHEMISTRY; METALLURGY
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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
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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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    • 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
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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
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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)
    • C12Y305/04002—Adenine deaminase (3.5.4.2)
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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)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/90—Fusion polypeptide containing a motif for post-translational modification
    • C07K2319/92—Fusion polypeptide containing a motif for post-translational modification containing an intein ("protein splicing")domain
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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]
    • C—CHEMISTRY; METALLURGY
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3112011A 2018-09-07 2019-09-07 Compositions and methods for delivering a nucleobase editing system Pending CA3112011A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862728703P 2018-09-07 2018-09-07
US62/728,703 2018-09-07
US201862779404P 2018-12-13 2018-12-13
US62/779,404 2018-12-13
PCT/US2019/050111 WO2020051561A1 (en) 2018-09-07 2019-09-07 Compositions and methods for delivering a nucleobase editing system

Publications (1)

Publication Number Publication Date
CA3112011A1 true CA3112011A1 (en) 2020-03-12

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Application Number Title Priority Date Filing Date
CA3112011A Pending CA3112011A1 (en) 2018-09-07 2019-09-07 Compositions and methods for delivering a nucleobase editing system

Country Status (8)

Country Link
US (1) US12529041B2 (https=)
EP (1) EP3847254A4 (https=)
JP (2) JP7657711B2 (https=)
KR (1) KR20210055733A (https=)
CN (1) CN112969790B (https=)
AU (1) AU2019336245B2 (https=)
CA (1) CA3112011A1 (https=)
WO (1) WO2020051561A1 (https=)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11649442B2 (en) 2017-09-08 2023-05-16 The Regents Of The University Of California RNA-guided endonuclease fusion polypeptides and methods of use thereof
CN121555430A (zh) 2018-05-11 2026-02-24 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
WO2020168122A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
WO2020252455A1 (en) 2019-06-13 2020-12-17 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
WO2021016075A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
IL296024A (en) 2020-03-04 2022-10-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for modulating a genome
AU2021236683A1 (en) 2020-03-19 2022-11-17 Intellia Therapeutics, Inc. Methods and compositions for directed genome editing
GB2632565B (en) * 2020-04-09 2025-06-04 Verve Therapeutics Inc Base editing of PCSK9 and methods of using same for treatment of disease
EP4185600A4 (en) 2020-07-24 2024-12-04 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
US20230383277A1 (en) * 2020-10-14 2023-11-30 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
WO2023010135A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
EP4377459A2 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
JP2024533311A (ja) 2021-09-08 2024-09-12 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー ゲノムを調節するための方法及び組成物
US20250223584A2 (en) 2021-12-03 2025-07-10 The Broad Institute, Inc. Compositions and methods for efficient in vivo delivery
WO2023169454A1 (zh) * 2022-03-08 2023-09-14 中国科学院遗传与发育生物学研究所 腺嘌呤脱氨酶及其在碱基编辑中的用途
CN120303407A (zh) 2022-05-17 2025-07-11 恩维洛普治疗有限责任公司 用于有效体内递送的组合物和方法
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
EP4612166A1 (en) * 2022-11-01 2025-09-10 Memorial Sloan-Kettering Cancer Center Intein-based sorting system and modular chimeric polypeptides
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024233941A1 (en) * 2023-05-11 2024-11-14 Massachusetts Eye And Ear Infirmary Base editing approaches to treat abca4-associated stargardt disease
US12311033B2 (en) 2023-05-31 2025-05-27 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025038955A1 (en) * 2023-08-16 2025-02-20 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Systems and methods for improving safety of split intein aav mediated gene therapy
CN117568313B (zh) * 2024-01-15 2024-04-26 上海贝斯昂科生物科技有限公司 基因编辑组合物及其用途
WO2025194138A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome
WO2025194124A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. Modified st1cas9 guide nucleic acids
WO2025243196A1 (en) * 2024-05-21 2025-11-27 Fondazione Istituto Italiano Di Tecnologia Gene regulation system (tuser)
WO2026064753A1 (en) 2024-09-23 2026-03-26 Tune Therapeutics, Inc. Dna methyltransferase-like protein (dnmt3l) or dna methyltransferase 3a (dnmt3a) repressor systems for epigenetic editing

Family Cites Families (197)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH0329734A (ja) 1989-06-20 1991-02-07 Kubota Corp ラベルプリンタ
CA2392490A1 (en) 1999-11-24 2001-05-31 Mcs Micro Carrier Systems Gmbh 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
US20040115184A1 (en) 2001-02-27 2004-06-17 Smith Harold C 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
US20130011380A1 (en) 2009-12-18 2013-01-10 Blau Helen M Use of Cytidine Deaminase-Related Agents to Promote Demethylation and Cell Reprogramming
US20130109048A1 (en) 2010-07-09 2013-05-02 Ecole Polytechnique Federale De Lausanne (Epfl) Method for in-vitro monitoring of neuronal disorders and use thereof
EP2761006B1 (en) 2011-09-28 2016-12-14 Zera Intein Protein Solutions, S.L. Split inteins and uses thereof
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
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US20150017136A1 (en) 2013-07-15 2015-01-15 Cellectis Methods for engineering allogeneic and highly active t cell for immunotherapy
US9102936B2 (en) 2012-06-11 2015-08-11 Agilent Technologies, Inc. Method of adaptor-dimer subtraction using a CRISPR CAS6 protein
DK3431497T3 (da) 2012-06-27 2022-10-10 Univ Princeton Spaltede inteiner, konjugater og anvendelser deraf
EP3363902B1 (en) 2012-12-06 2019-11-27 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES 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
PL3546572T3 (pl) 2013-05-13 2024-07-22 Cellectis Chimeryczny receptor antygenowy swoisty względem cd19 i jego zastosowania
WO2014186686A2 (en) 2013-05-17 2014-11-20 Two Blades Foundation Targeted mutagenesis and genome engineering in plants using rna-guided cas nucleases
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
EP3004370B1 (en) 2013-06-05 2024-08-21 Duke University Rna-guided gene editing and gene regulation
JP2016528890A (ja) 2013-07-09 2016-09-23 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9994831B2 (en) 2013-12-12 2018-06-12 The Regents Of The University Of California Methods and compositions for modifying a single stranded target nucleic acid
AU2014361781B2 (en) 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
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
SG11201609211VA (en) * 2014-03-05 2016-12-29 Nat Univ Corp Univ Kobe Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
EP3593812A3 (en) 2014-03-15 2020-05-27 Novartis AG Treatment of cancer using chimeric antigen receptor
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
AU2015289644A1 (en) 2014-07-15 2017-02-02 Juno Therapeutics, Inc. Engineered cells for adoptive cell therapy
WO2016022363A2 (en) * 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
JP2017522893A (ja) 2014-07-31 2017-08-17 セレクティスＣｅｌｌｅｃｔｉｓ Ｒｏｒ１特異的多重鎖キメラ抗原受容体
EP3633032A3 (en) 2014-08-28 2020-07-29 North Carolina State University Novel cas9 proteins and guiding features for dna targeting and genome editing
ES2753391T3 (es) 2014-10-14 2020-04-08 Halozyme Inc Composiciones de adenosina desaminasa 2 (ADA2), variantes de la misma y métodos de uso de las mismas
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
US10920215B2 (en) 2014-11-04 2021-02-16 National University Corporation Kobe University Method for modifying genome sequence to introduce specific mutation to targeted DNA sequence by base-removal reaction, and molecular complex used therein
MA40921A (fr) 2014-11-05 2017-09-12 Abbvie Stemcentrx Llc Récepteurs antigéniques chimériques anti-cldn et procédés d'utilisation
US11072644B2 (en) 2014-11-12 2021-07-27 Allogene Therapeutics, Inc. Inhibitory chimeric antigen receptors
EA036379B1 (ru) 2014-12-12 2020-11-02 Блубёрд Био, Инк. Химерные антигенные рецепторы к bcma
US10676737B2 (en) 2014-12-17 2020-06-09 Proqr Therapeutics Ii B.V. Targeted RNA editing
WO2016112242A1 (en) 2015-01-08 2016-07-14 President And Fellows Of Harvard College Split cas9 proteins
MA41613A (fr) 2015-02-23 2018-01-02 Abbvie Stemcentrx Llc Récepteurs antigéniques chimériques anti-dll3 et procédés d'utilisation desdits récepteurs
EP3265559B1 (en) 2015-03-03 2021-01-06 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
KR20170137079A (ko) 2015-03-11 2017-12-12 셀렉티스 그것들의 지속을 증가시키기 위한 동종이계 t 세포의 조작 및/또는 환자들 내로 이식 방법들
CN106011104B (zh) * 2015-05-21 2019-09-27 清华大学 利用拆分Cas系统进行基因编辑和表达调控方法
JP6949728B2 (ja) 2015-05-29 2021-10-13 ジュノー セラピューティクス インコーポレイテッド 遺伝子操作された細胞における阻害相互作用を調節するための組成物および方法
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2016205759A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
US11479793B2 (en) 2015-07-15 2022-10-25 Rutgers, The State University Of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US20180258149A1 (en) 2015-09-17 2018-09-13 Novartis Ag Car t cell therapies with enhanced efficacy
WO2017048969A1 (en) 2015-09-17 2017-03-23 The Regents Of The University Of California Variant cas9 polypeptides comprising internal insertions
JP7109784B2 (ja) 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したＣａｓ９蛋白質
MX2018005618A (es) 2015-11-05 2018-08-01 Juno Therapeutics Inc Receptores quimericos que contienen dominios inductores de traf y composiciones y metodos 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
CA2999649A1 (en) 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a
CA3006618A1 (en) 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
ES2949163T3 (es) 2016-01-29 2023-09-26 Univ Princeton Inteínas divididas con actividad de corte y empalme excepcional
WO2017165862A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
TWI773666B (zh) 2016-03-30 2022-08-11 美商英特利亞醫療公司 Crispr/cas 組分之脂質奈米粒子調配物
WO2017180993A1 (en) 2016-04-14 2017-10-19 Bluebird Bio, Inc. Salvage chimeric antigen receptor systems
CN110382692A (zh) 2016-04-19 2019-10-25 博德研究所 新型crispr酶以及系统
US20200263190A1 (en) 2016-04-19 2020-08-20 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018020323A2 (en) 2016-07-25 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of fatty acid disorders
NZ750256A (en) 2016-08-03 2026-02-27 Dipersio John F Gene editing of car-t cells for the treatment of t cell malignancies with chimeric antigen receptors
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018035388A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018035503A1 (en) 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
US10941402B2 (en) 2016-09-01 2021-03-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
KR20240005168A (ko) 2016-11-04 2024-01-11 2세븐티 바이오, 인코포레이티드 항-bcma car t 세포 조성물
WO2018089664A1 (en) 2016-11-11 2018-05-17 The Regents Of The University Of California Variant rna-guided polypeptides and methods of use
CN107043779B (zh) 2016-12-01 2020-05-12 中国农业科学院作物科学研究所 一种CRISPR/nCas9介导的定点碱基替换在植物中的应用
AU2017374044B2 (en) 2016-12-08 2023-11-30 Intellia Therapeutics, Inc. Modified guide RNAs
AU2017379073B2 (en) 2016-12-22 2023-12-14 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
EP3559223A1 (en) 2016-12-23 2019-10-30 President and Fellows of Harvard College Gene editing of pcsk9
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018129129A1 (en) 2017-01-05 2018-07-12 Rutgers, The State University Of New Jersey Targeted gene editing platform independent of dna double strand break and uses thereof
WO2018152197A1 (en) 2017-02-15 2018-08-23 Massachusetts Institute Of Technology Dna writers, molecular recorders and uses thereof
SG11201907870VA (en) 2017-02-28 2019-09-27 Vor Biopharma Inc Compositions and methods for inhibition of lineage specific proteins
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
CN106916852B (zh) 2017-04-13 2020-12-04 上海科技大学 一种碱基编辑系统及其构建和应用方法
WO2018195545A2 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2018213708A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
US20200172895A1 (en) 2017-05-25 2020-06-04 The General Hospital Corporation Using split deaminases to limit unwanted off-target base editor deamination
CA3065946A1 (en) 2017-06-05 2018-12-13 Research Institute At Nationwide Children's Hospital Enhanced modified viral capsid proteins
AU2018290843B2 (en) 2017-06-26 2025-04-24 Massachusetts Institute Of Technology CRISPR/Cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing
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
US11168322B2 (en) 2017-06-30 2021-11-09 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
CN118530967A (zh) 2017-08-23 2024-08-23 通用医疗公司 具有改变的PAM特异性的工程化的CRISPR-Cas9核酸酶
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
EP4389889A3 (en) 2017-10-06 2024-09-04 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
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
WO2019183000A1 (en) 2018-03-19 2019-09-26 University Of Massachusetts Modified guide rnas for crispr genome editing
CN121555430A (zh) 2018-05-11 2026-02-24 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
JP7558929B2 (ja) 2018-05-11 2024-10-01 ビーム セラピューティクス インク． プログラム可能塩基エディターシステムを用いて病原性変異を抑制する方法
AU2019266327B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of editing single nucleotide polymorphism using programmable base editor systems
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
AU2019316094B2 (en) 2018-08-03 2026-02-19 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
US11530245B2 (en) 2018-08-31 2022-12-20 Washington University Split intein mediated polymerization and production of mussel foot adhesive protein materials
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
CN109295186B (zh) 2018-09-30 2023-10-03 中山大学 一种基于全基因组测序检测腺嘌呤单碱基编辑系统脱靶效应的方法及其在基因编辑中的应用
AU2019360372B2 (en) 2018-10-15 2026-02-26 Fondazione Telethon Intein proteins and uses thereof
US20220023348A1 (en) 2018-11-28 2022-01-27 Forty Seven, Inc. Genetically modified hspcs resistant to ablation regime
US20220047637A1 (en) 2018-11-29 2022-02-17 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US11667682B2 (en) 2018-12-13 2023-06-06 Washington University Split intein mediated protein polymerization for microbial production of materials
WO2020146627A1 (en) 2019-01-10 2020-07-16 California Institute Of Technology A synthetic system for tunable thresholding of protein signals
EP3918077A4 (en) 2019-01-31 2023-03-29 Beam Therapeutics, Inc. NUCLEOBASE EDITORS WITH REDUCED OFF-TARGET DEAMINATION AND METHODS OF USING THEM TO MODIFY A NUCLEOBASE TARGET SEQUENCE
KR20210121113A (ko) 2019-01-31 2021-10-07 빔 테라퓨틱스, 인크. 비-표적 탈아미노화가 감소된 핵염기 편집기 및 핵염기 편집기를 특성규명하기 위한 분석
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
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
WO2020168122A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
KR20210126680A (ko) 2019-02-13 2021-10-20 빔 테라퓨틱스, 인크. 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법
US20220235347A1 (en) 2019-02-13 2022-07-28 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
AU2020223306A1 (en) 2019-02-13 2021-08-05 Beam Therapeutics Inc. Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
KR20210139265A (ko) 2019-02-13 2021-11-22 빔 테라퓨틱스, 인크. 표적 서열에서 핵염기를 변형하기 위한 아데노신 데아미나제 염기 편집기 및 이의 사용 방법
US20220127594A1 (en) 2019-02-13 2022-04-28 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
KR102129377B1 (ko) 2019-02-21 2020-07-02 성균관대학교산학협력단 특정 특성이 발현된 단백질을 암호화하는 플라스미드를 선별하는 유도 플라스미드 디스플레이 시스템
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020231863A1 (en) 2019-05-10 2020-11-19 Beam Therapeutics Inc. Compositions and methods for treating hepatitis b
WO2020236982A1 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Aav delivery of nucleobase editors
US20220387622A1 (en) 2019-05-21 2022-12-08 Beam Therapeutics Inc. Methods of editing a single nucleotide polymorphism using programmable base editor systems
WO2020252455A1 (en) 2019-06-13 2020-12-17 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
WO2021016075A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
AU2020322021A1 (en) 2019-07-30 2022-02-10 Pairwise Plants Services, Inc. Morphogenic regulators and methods of using the same
KR102258713B1 (ko) 2019-07-31 2021-05-31 한양대학교 산학협력단 사이토신 염기교정용 조성물 및 이의 용도
CA3152861A1 (en) 2019-08-29 2021-03-04 Beam Therapeutics Inc. Compositions and methods for editing a mutation to permit transcription or expression
WO2021042062A2 (en) 2019-08-30 2021-03-04 Joung J Keith Combinatorial adenine and cytosine dna base editors
CN114667149A (zh) 2019-09-09 2022-06-24 比姆医疗股份有限公司 新型核碱基编辑器及其使用方法
JP7814052B2 (ja) 2019-09-17 2026-02-16 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 標的ゲノム修飾のための高度に効率的なｒｎａ－アプタマー動員媒介性ｄｎａ塩基エディターおよびそれらの使用
US20220372496A1 (en) 2019-09-24 2022-11-24 MVRIX Co., Ltd. Nano-perforator having improved anti-viral activity
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
US11591607B2 (en) 2019-10-24 2023-02-28 Pairwise Plants Services, Inc. Optimized CRISPR-Cas nucleases and base editors and methods of use thereof
KR20220110739A (ko) 2019-10-30 2022-08-09 페어와이즈 플랜츠 서비시즈, 인크. 유형 v crispr-cas 염기 편집제 및 그의 사용 방법
CA3162499A1 (en) 2019-11-22 2021-05-27 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
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
GB201918693D0 (en) 2019-12-18 2020-01-29 Univ Southampton Peptide
WO2021158921A2 (en) 2020-02-05 2021-08-12 The Broad Institute, Inc. Adenine base editors and uses thereof
JP2023516692A (ja) 2020-03-04 2023-04-20 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー ゲノムを調節するための方法及び組成物
CA3174483A1 (en) 2020-03-04 2021-09-10 Flagship Pioneering Innovations Vi, Llc Improved methods and compositions for modulating a genome
IL296024A (en) 2020-03-04 2022-10-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for modulating a genome
AU2021232069A1 (en) 2020-03-05 2022-11-03 Flagship Pioneering Innovations Vi, Llc Host defense suppressing methods and compositions for modulating a genome
AU2021236683A1 (en) 2020-03-19 2022-11-17 Intellia Therapeutics, Inc. Methods and compositions for directed genome editing
EP3885440A1 (en) 2020-03-26 2021-09-29 Splicebio, S.L. Split inteins and their uses
WO2021207651A2 (en) 2020-04-09 2021-10-14 Verve Therapeutics, Inc. Chemically modified guide rnas for genome editing with cas12b
WO2021209574A1 (en) 2020-04-15 2021-10-21 Fondazione Telethon Constructs comprising inteins
WO2021222318A1 (en) 2020-04-28 2021-11-04 The Broad Institute, Inc. Targeted base editing of the ush2a gene
EP4179079A1 (en) 2020-07-10 2023-05-17 Horizon Discovery Limited Method for producing genetically modified cells
JP2023534693A (ja) 2020-07-15 2023-08-10 ライフエディット セラピューティクス，インコーポレイティド ウラシル安定化タンパク質並びにその活性断片及びバリアント並びに使用方法
EP4185600A4 (en) 2020-07-24 2024-12-04 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
CA3192224A1 (en) 2020-09-11 2022-03-17 Jyun-Liang LIN Base editing enzymes
TWI910226B (zh) 2020-09-11 2026-01-01 美商生命編輯治療學公司 Ｄｎａ修飾酶及活性片段，及其變異體與使用方法
US20230383277A1 (en) 2020-10-14 2023-11-30 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
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
EP4274897A4 (en) 2021-01-08 2025-03-05 The General Hospital Corporation GENOMEDITING APPROACHES TO THE TREATMENT OF SPINAL MUSCLE ATROPHY
WO2022178307A1 (en) 2021-02-19 2022-08-25 Beam Therapeutics Inc. Recombinant rabies viruses for gene therapy
CA3214494A1 (en) 2021-03-26 2022-09-29 Beam Therapeutics Inc. Adenosine deaminase variants and uses thereof
CN113813398B (zh) 2021-04-23 2023-11-07 天津医科大学 一种向表面富含神经节苷脂的细胞递送重组蛋白质的方法
US20240327859A1 (en) 2021-07-02 2024-10-03 University Of Maryland, College Park Cytidine deaminases and methods of genome editing using the same
AU2022311013A1 (en) 2021-07-16 2024-02-08 President And Fellows Of Harvard College Context-specific adenine base editors and uses thereof
CN113480613B (zh) 2021-08-04 2022-04-15 江南大学 一种自剪切蛋白标签、改造方法及其应用
CN113735941B (zh) 2021-08-13 2024-01-30 江南大学 一种蛋白纯化方法及其应用
US20240352439A1 (en) 2021-09-03 2024-10-24 The University Of Chicago Polypeptides and methods for modifying nucleic acids
AU2022349449A1 (en) 2021-09-22 2024-04-18 Verve Therapeutics, Inc. Gene editing of pcsk9 or angptl3 and compositions and methods of using same for treatment of disease
WO2023047338A1 (en) 2021-09-24 2023-03-30 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
CN117925585A (zh) 2022-04-07 2024-04-26 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
WO2023227669A2 (en) 2022-05-26 2023-11-30 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
CA3263821A1 (en) 2022-08-16 2024-02-22 The Broad Institute, Inc. ADVANCED CYTOSIN DESAMINASE AND DNA EDITING METHODS USING IT
CN119998447A (zh) 2022-09-23 2025-05-13 基础科学研究院 新型腺嘌呤脱氨酶变体及使用其进行碱基编辑的方法
CA3267375A1 (en) 2022-09-26 2024-04-04 Beam Therapeutics Inc. SYNTHETIC POLYPEPTIDES AND THEIR USES
WO2024179426A2 (en) 2023-02-28 2024-09-06 Accuredit Therapeutics (Suzhou) Co., Ltd. Deaminases for use in base editing
WO2024226156A1 (en) 2023-04-27 2024-10-31 University Of Massachusetts Cas-embedded cytidine deaminase ribonucleoprotein complexes having improved base editing specificity and efficiency
WO2024227047A2 (en) 2023-04-28 2024-10-31 Beam Therapeutics Inc. Modified guide rna
CN121889499A (zh) 2023-06-15 2026-04-17 比姆医疗股份有限公司 用于治疗α1-抗胰蛋白酶缺乏症的方法和组合物
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

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