JP2023518395A - 指向性ゲノム編集のための方法及び組成物 - Google Patents

指向性ゲノム編集のための方法及び組成物 Download PDF

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JP2023518395A
JP2023518395A JP2022555909A JP2022555909A JP2023518395A JP 2023518395 A JP2023518395 A JP 2023518395A JP 2022555909 A JP2022555909 A JP 2022555909A JP 2022555909 A JP2022555909 A JP 2022555909A JP 2023518395 A JP2023518395 A JP 2023518395A
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nucleic acid
reverse transcriptase
composition
region
guide nucleic
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オマー ハルペリン，シャクド
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インテリア セラピューティクス，インコーポレイテッド
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JP2022555909A 2020-03-19 2021-03-18 指向性ゲノム編集のための方法及び組成物 Pending JP2023518395A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202062992032P 2020-03-19 2020-03-19
US62/992,032 2020-03-19
US202063055829P 2020-07-23 2020-07-23
US63/055,829 2020-07-23
US202163153161P 2021-02-24 2021-02-24
US63/153,161 2021-02-24
PCT/US2021/023041 WO2021188840A1 (en) 2020-03-19 2021-03-18 Methods and compositions for directed genome editing

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JP2023518395A true JP2023518395A (ja) 2023-05-01
JP2023518395A5 JP2023518395A5 (https=) 2024-03-29

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EP (1) EP4121522A4 (https=)
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KR (1) KR20230003467A (https=)
CN (1) CN115667505A (https=)
AU (1) AU2021236683A1 (https=)
MX (1) MX2022011562A (https=)
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Families Citing this family (55)

* 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
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
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
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
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
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
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
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
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
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
IL297761A (en) * 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
CA3189601A1 (en) 2020-07-24 2022-01-27 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
EP4232583A1 (en) 2020-10-21 2023-08-30 Massachusetts Institute of Technology Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)
US20240026381A1 (en) * 2020-11-03 2024-01-25 The Board Of Trustees Of The University Of Illinois Split prime editing platforms
WO2023288332A2 (en) * 2021-07-16 2023-01-19 Prime Medicine, Inc. Genome editing compositions and methods for treatment of wilson's disease
EP4392039A4 (en) * 2021-08-24 2025-10-15 Prime Medicine Inc GENOME EDITING COMPOSITIONS AND METHODS FOR TREATING RETINOPATHY
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
EP4413128A1 (en) * 2021-10-08 2024-08-14 The General Hospital Corporation Improved crispr prime editors
EP4419688A2 (en) * 2021-10-21 2024-08-28 Prime Medicine, Inc. Genome editing compositions and methods for treatment of usher syndrome type 3
JP2024543481A (ja) * 2021-11-12 2024-11-21 リプレイス セラピューティクス リミテッド ライアビリティ カンパニー 直接置き換えゲノム編集
US11866728B2 (en) 2022-01-21 2024-01-09 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
AU2023293131A1 (en) 2022-06-16 2024-12-12 Intellia Therapeutics, Inc. Compositions and methods for reducing mhc class i in a cell
CN115029374B (zh) * 2022-06-24 2023-12-26 安徽省农业科学院水稻研究所 一种用于骨干载体的pegRNA表达框及相应骨干载体和应用
JP2025525569A (ja) 2022-07-18 2025-08-05 レナゲード セラピューティクス マネージメント インコーポレイテッド 遺伝子編集成分、システム、及び使用方法
CA3265664A1 (en) 2022-08-25 2024-02-29 Life Edit Therapeutics, Inc. CHEMICAL MODIFICATION OF GUIDE RNA WITH A LOCKED NUCLEIC ACID FOR RNA-GUIDED NUCLEASE-MEDIATED GENE EDITING
KR20250126155A (ko) 2022-11-04 2025-08-22 라이프 에디트 테라퓨틱스, 인크. 내부 삽입 부위를 갖는 진화된 아데닌 데아미나제 및 rna-가이드 뉴클레아제 융합 단백질 및 사용 방법
EP4634383A1 (en) 2022-12-16 2025-10-22 Life Edit Therapeutics, Inc. Guide rnas that target foxp3 gene and methods of use
WO2024127370A1 (en) 2022-12-16 2024-06-20 LifeEDIT Therapeutics, Inc. Guide rnas that target trac gene and methods of use
WO2024178397A2 (en) 2023-02-24 2024-08-29 Elevatebio Technologies, Inc. Modified immune effector cells and methods of use
WO2025022367A2 (en) 2023-07-27 2025-01-30 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2025038637A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
IL326191A (en) 2023-08-14 2026-03-01 Intellia Therapeutics Inc Compositions and methods for genetic modification of transforming growth factor beta receptor type 2 (tgfβr2)
WO2025038646A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Cd70 car-t compositions and methods for cell-based therapy
WO2025038642A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying cd70
AU2024335327A1 (en) 2023-09-01 2026-03-26 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025076306A1 (en) * 2023-10-06 2025-04-10 University Of Massachusetts Prime editors having improved prime editing efficiency
WO2025081111A1 (en) * 2023-10-12 2025-04-17 The University Of Chicago Methods and compositions for characterizing rna-binding protein binding sites by in-situ reverse transcription-based sequencing
WO2025083619A1 (en) 2023-10-18 2025-04-24 Life Edit Therapeutics, Inc. Rna-guided nucleases and acive fragments and variants thereof and methods of use
WO2025106766A1 (en) * 2023-11-15 2025-05-22 Yale University Fusion-modified enzyme constructs
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025174908A1 (en) 2024-02-12 2025-08-21 Life Edit Therapeutics, Inc. Novel rna-guided nucleases and proteins for polymerase editing
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025226730A1 (en) * 2024-04-22 2025-10-30 Transit Therapeutics, Inc. Revision of genetic material using direct replacement editing
WO2025235506A2 (en) * 2024-05-06 2025-11-13 Flagship Pioneering Innovations Vi, Llc Atp7b-modulating compositions and methods
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy
WO2025264736A1 (en) * 2024-06-18 2025-12-26 Blake Richards Logic capable protease probes and uses thereof
WO2026003754A1 (en) 2024-06-25 2026-01-02 Life Edit Therapeutics, Inc. Novel reverse transcriptases and uses thereof

Family Cites Families (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69923274T2 (de) * 1998-10-12 2006-03-30 Roche Diagnostics Gmbh Deoxynukleotidtriphosphatkinase aus Insektenzellen zur Synthese von Nukleosidmonophosphaten
GB0806562D0 (en) * 2008-04-10 2008-05-14 Fermentas Uab Production of nucleic acid
WO2011116184A2 (en) 2010-03-17 2011-09-22 Cornell University Methods and compositions for targeted mutagenesis in bacteria
FR2973040B1 (fr) * 2011-03-23 2016-04-15 Centre Nat De La Rech Scient (C N R S) Methode de criblage de composes antiretroviraux et vaccin
US9161995B2 (en) 2011-07-25 2015-10-20 Sangamo Biosciences, Inc. Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (CFTR) gene
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
RU2662932C2 (ru) * 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
WO2014186686A2 (en) 2013-05-17 2014-11-20 Two Blades Foundation Targeted mutagenesis and genome engineering in plants using rna-guided cas nucleases
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
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US9963687B2 (en) 2014-08-27 2018-05-08 New England Biolabs, Inc. Fusion polymerase and method for using the same
EP3188763B1 (en) 2014-09-02 2020-05-13 The Regents of The University of California Methods and compositions for rna-directed target dna modification
EP3201340B1 (en) * 2014-10-01 2020-12-02 The General Hospital Corporation Methods for increasing efficiency of nuclease-induced homology-directed repair
CN107614680A (zh) 2015-05-14 2018-01-19 南加利福尼亚大学 利用重组核酸内切酶系统的最佳化基因编辑
CA3001683C (en) 2015-06-05 2024-06-04 The Regents Of The University Of California Methods and compositions for generating crispr/cas guide rnas
LT3505174T (lt) * 2015-06-17 2020-09-10 The Trustees Of Columbia University In The City Of New York Deoksinukleozidų terapija, skirta gydyti ligas, kurias sukelia nesubalansuoti nukleotidų kiekiai, įskaitant mitochondrinės dnr išsekimo sindromus
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
EP3163302A1 (en) * 2015-11-02 2017-05-03 Johann Wolfgang Goethe-Universität Frankfurt am Main Samhd1 modulation for treating resistance to cancer therapy
WO2017106616A1 (en) 2015-12-17 2017-06-22 The Regents Of The University Of Colorado, A Body Corporate Varicella zoster virus encoding regulatable cas9 nuclease
US11447768B2 (en) 2016-03-01 2022-09-20 University Of Florida Research Foundation, Incorporated Molecular cell diary system
EP3426353A4 (en) * 2016-03-09 2020-05-06 The University of Massachusetts USE OF MODIFIED HIV-1 TO GENERATE FULLY HUMAN ANTIBODIES
AU2017257274B2 (en) 2016-04-19 2023-07-13 Massachusetts Institute Of Technology Novel CRISPR enzymes and systems
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
ES2985812T3 (es) * 2016-09-09 2024-11-07 The Board Of Trustees Of The Leland Stanfordjunior Univ Edición genómica de precisión de alto rendimiento
KR20190098747A (ko) * 2016-12-05 2019-08-22 주노 쎄러퓨티크스 인코퍼레이티드 입양 세포 치료법을 위한 조작된 세포의 제조방법
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
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
WO2019070843A1 (en) * 2017-10-03 2019-04-11 Board Of Regents, The University Of Texas System INVERSE TRANSCRIPTASE WITH LTR-FREE BACKLIGHTS AND USES THEREOF
CA3105925A1 (en) * 2018-07-10 2020-01-16 Alia Therapeutics S.R.L. Vesicles for traceless delivery of guide rna molecules and/or guide rna molecule/rna-guided nuclease complex(es) and a production method thereof
EP3844272A1 (en) * 2018-08-28 2021-07-07 Flagship Pioneering Innovations VI, LLC Methods and compositions for modulating a genome
EP3847254A4 (en) * 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
WO2020191233A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
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

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
NATURE, vol. 576, JPN6025037485, 2019, pages 149 - 157, ISSN: 0005833926 *
THE JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 280, no. 13, JPN6025037484, 2005, pages 12190 - 12200, ISSN: 0005833927 *

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