JP2019536464A5 - - Google Patents

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JP2019536464A5
JP2019536464A5 JP2019530394A JP2019530394A JP2019536464A5 JP 2019536464 A5 JP2019536464 A5 JP 2019536464A5 JP 2019530394 A JP2019530394 A JP 2019530394A JP 2019530394 A JP2019530394 A JP 2019530394A JP 2019536464 A5 JP2019536464 A5 JP 2019536464A5
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nucleotides
sgrna
modified
region
modification
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JP2019530394A
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JP2019536464A (ja
JP7696694B2 (ja
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Priority claimed from PCT/US2017/065306 external-priority patent/WO2018107028A1/en
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Priority to JP2022096277A priority Critical patent/JP7625555B2/ja
Priority to JP2024158151A priority patent/JP2024178237A/ja
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JP2019530394A 2016-12-08 2017-12-08 修飾されたガイドrna Active JP7696694B2 (ja)

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JP2022096277A JP7625555B2 (ja) 2016-12-08 2022-06-15 修飾されたガイドrna
JP2024158151A JP2024178237A (ja) 2016-12-08 2024-09-12 修飾されたガイドrna

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US201662431756P 2016-12-08 2016-12-08
US62/431,756 2016-12-08
PCT/US2017/065306 WO2018107028A1 (en) 2016-12-08 2017-12-08 Modified guide rnas

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JP2019536464A JP2019536464A (ja) 2019-12-19
JP2019536464A5 true JP2019536464A5 (enExample) 2021-01-21
JP7696694B2 JP7696694B2 (ja) 2025-06-23

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JP2022096277A Active JP7625555B2 (ja) 2016-12-08 2022-06-15 修飾されたガイドrna
JP2024158151A Pending JP2024178237A (ja) 2016-12-08 2024-09-12 修飾されたガイドrna

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JP2024158151A Pending JP2024178237A (ja) 2016-12-08 2024-09-12 修飾されたガイドrna

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US (3) US11479767B2 (enExample)
EP (1) EP3551757A1 (enExample)
JP (3) JP7696694B2 (enExample)
KR (1) KR102595683B1 (enExample)
CN (2) CN110291198B (enExample)
AU (2) AU2017374044B2 (enExample)
BR (1) BR112019011509A2 (enExample)
CA (1) CA3046376A1 (enExample)
CO (1) CO2019007258A2 (enExample)
EA (1) EA201991369A1 (enExample)
IL (1) IL267024B2 (enExample)
MX (2) MX2024001138A (enExample)
MY (1) MY206324A (enExample)
PH (1) PH12019501262A1 (enExample)
SG (1) SG10202106058WA (enExample)
TW (1) TWI835719B (enExample)
WO (1) WO2018107028A1 (enExample)
ZA (1) ZA202007633B (enExample)

Families Citing this family (133)

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Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US11279928B2 (en) 2015-06-29 2022-03-22 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
US11845933B2 (en) 2016-02-03 2023-12-19 Massachusetts Institute Of Technology Structure-guided chemical modification of guide RNA and its applications
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
KR102551664B1 (ko) * 2016-12-22 2023-07-05 인텔리아 테라퓨틱스, 인크. 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
KR102712656B1 (ko) 2017-01-23 2024-10-04 리제너론 파마슈티칼스 인코포레이티드 Hsd17b13 변종 및 이것의 용도
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11479802B2 (en) 2017-04-11 2022-10-25 Regeneron Pharmaceuticals, Inc. Assays for screening activity of modulators of members of the hydroxy steroid (17-beta) dehydrogenase (HSD17B) family
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
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
KR102780441B1 (ko) 2017-07-31 2025-03-17 리제너론 파마슈티칼스 인코포레이티드 Cas-형질전환 마우스 배아 줄기 세포 및 마우스 및 이것의 용도
KR20200033259A (ko) 2017-07-31 2020-03-27 리제너론 파마슈티칼스 인코포레이티드 생체내에서 CRISPR/Cas-매개된 파괴 또는 삭제 및 외인성 도너 핵산과의 CRISPR/Cas-유도된 재조합을 평가하기 위한 방법 및 조성물
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
WO2019067872A1 (en) 2017-09-29 2019-04-04 Intellia Therapeutics, Inc. COMPOSITIONS AND METHODS FOR EDITING THE TTR GENE AND TREATING AMYLOID DOSE ATTR
EP3585162B1 (en) 2017-09-29 2023-08-30 Regeneron Pharmaceuticals, Inc. Rodents comprising a humanized ttr locus and methods of use
AU2018348195B2 (en) 2017-10-11 2025-05-15 Regeneron Pharmaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
US20190233816A1 (en) * 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
CN111885915B (zh) 2018-03-19 2023-04-28 瑞泽恩制药公司 使用crispr/cas系统对动物进行转录调制
BR112020018758A2 (pt) 2018-03-21 2021-01-26 Regeneron Pharmaceuticals, Inc. agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios
US12133884B2 (en) 2018-05-11 2024-11-05 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
CA3113190A1 (en) 2018-07-31 2020-02-06 Intellia Therapeutics, Inc. Compositions and methods for hydroxyacid oxidase 1 (hao1) gene editing for treating primary hyperoxaluria type 1 (ph1)
WO2020051283A1 (en) 2018-09-05 2020-03-12 The Regents Of The University Of California Generation of heritably gene-edited plants without tissue culture
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
BR112021005718A2 (pt) 2018-09-28 2021-06-22 Intellia Therapeutics, Inc. composições e métodos para edição de genes de lactato desidrogenase (ldha)
AU2019362879A1 (en) 2018-10-16 2021-05-27 Intellia Therapeutics, Inc. Compositions and methods for immunotherapy
BR112021007301A2 (pt) 2018-10-18 2021-07-27 Intellia Therapeutics, Inc. composições e métodos para expressar fator ix
CN113195721A (zh) 2018-10-18 2021-07-30 英特利亚治疗股份有限公司 治疗α-1抗胰蛋白酶缺乏症的组合物和方法
EP3867381A2 (en) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for transgene expression from an albumin locus
US12281338B2 (en) 2018-10-29 2025-04-22 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
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
CN118064502A (zh) 2019-04-03 2024-05-24 瑞泽恩制药公司 用于将抗体编码序列插入到安全港基因座中的方法和组合物
ES2923629T3 (es) 2019-04-04 2022-09-29 Regeneron Pharma Métodos para la introducción sin cicatrices de modificaciones dirigidas en vectores de direccionamiento
AU2020253532B2 (en) 2019-04-04 2024-06-20 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized coagulation factor 12 locus
CN113966401A (zh) 2019-04-10 2022-01-21 犹他大学研究基金会 用于治疗amd的htra1调节
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12365921B2 (en) 2019-05-30 2025-07-22 National University Corporation Hokkaido University Lipid nanoparticle
WO2020247452A1 (en) 2019-06-04 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use
SG11202111256XA (en) 2019-06-07 2021-11-29 Regeneron Pharma Non-human animals comprising a humanized albumin locus
CN114258398A (zh) 2019-06-13 2022-03-29 总医院公司 工程化的人内源性病毒样颗粒及使用其递送至细胞的方法
WO2021050940A1 (en) 2019-09-13 2021-03-18 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4054651A1 (en) 2019-11-08 2022-09-14 Regeneron Pharmaceuticals, Inc. Crispr and aav strategies for x-linked juvenile retinoschisis therapy
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
CN115176001A (zh) * 2019-12-11 2022-10-11 因特利亚治疗公司 用于基因编辑的修饰的引导rna
CN115362256A (zh) 2020-02-07 2022-11-18 因特利亚治疗公司 用于激肽释放酶(klkb1)基因编辑的组合物和方法
AU2021230546A1 (en) 2020-03-04 2022-10-13 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
EP4125348A1 (en) 2020-03-23 2023-02-08 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
BR112022020407A2 (pt) * 2020-04-09 2023-05-02 Verve Therapeutics Inc Edição base do pcsk9 e métodos de uso do mesmo para tratamento de doenças
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート,インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
CA3177463A1 (en) * 2020-05-12 2021-11-18 Erik Joseph SONTHEIMER Modified guide rnas for crispr genome editing
BR112023001272A2 (pt) 2020-07-24 2023-04-04 Massachusetts Gen Hospital Partículas semelhantes a vírus aprimoradas e métodos de uso das mesmas para entrega às células
CN112111507B (zh) * 2020-08-10 2022-08-23 江苏大学 一种灰树花CRISPR-Cas9基因编辑系统、方法及应用
KR102493512B1 (ko) * 2020-10-08 2023-02-01 주식회사 진코어 CRISPR/Cas12a 시스템을 위한 엔지니어링 된 crRNA
WO2022081702A1 (en) * 2020-10-13 2022-04-21 The Trustees Of The University Of Pennsylvania In vivo targeting of cd4+-t cells for mrna therapeutics
US20220218622A1 (en) 2020-10-14 2022-07-14 George Mason Research Foundation, Inc. Ionizable lipids and methods of manufacture and use thereof
AU2021396403A1 (en) 2020-12-11 2023-06-29 Intellia Therapeutics, Inc. Compositions and methods for reducing mhc class ii in a cell
KR20230129996A (ko) 2020-12-11 2023-09-11 인텔리아 테라퓨틱스, 인크. 탈아미노화를 포함하는 게놈 편집을 위한 폴리뉴클레오타이드,조성물 및 방법
KR20230135068A (ko) 2020-12-23 2023-09-22 인텔리아 테라퓨틱스, 인크. 세포에서 ciita를 유전적으로 변형시키기 위한 조성물및 방법
AU2021409732A1 (en) 2020-12-23 2023-07-20 Intellia Therapeutics, Inc. Compositions and methods for reducing hla-a in a cell
MX2023007801A (es) 2020-12-30 2023-08-21 Intellia Therapeutics Inc Células t diseñadas.
WO2022170059A1 (en) 2021-02-05 2022-08-11 Christiana Care Health Services, Inc. Methods of and compositions for reducing gene expression and/or activity
EP4288088A2 (en) 2021-02-08 2023-12-13 Intellia Therapeutics, Inc. Lymphocyte activation gene 3 (lag3) compositions and methods for immunotherapy
JP2024505672A (ja) 2021-02-08 2024-02-07 インテリア セラピューティクス,インコーポレイテッド 免疫療法のためのナチュラルキラー細胞受容体2b4組成物及び方法
WO2022170193A2 (en) 2021-02-08 2022-08-11 Intellia Therapeutics, Inc. T-cell immunoglobulin and mucin domain 3 (tim3) compositions and methods for immunotherapy
KR102689529B1 (ko) * 2021-06-28 2024-07-29 서울대학교산학협력단 sgRNA와 dCas9 간의 결합을 조절하여 박테리아에서 유전자 발현을 정량적으로 조절할 수 있는 방법
JP2024534114A (ja) 2021-08-24 2024-09-18 インテリア セラピューティクス,インコーポレイテッド 細胞療法用のプログラム細胞死タンパク質1(pd1)組成物及び方法
CA3231677A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
AU2022345881A1 (en) 2021-09-20 2024-03-21 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
WO2023047338A1 (en) * 2021-09-24 2023-03-30 Crispr Therapeutics Ag PRODRUG INCORPORATED sgRNA SYNTHESIS
CN118251491A (zh) 2021-10-28 2024-06-25 瑞泽恩制药公司 用于敲除C5的CRISPR/Cas相关方法及组合物
EP4426836A1 (en) * 2021-11-03 2024-09-11 Intellia Therapeutics, Inc. Modified guide rnas for gene editing
CN118369110A (zh) 2021-11-03 2024-07-19 英特利亚治疗股份有限公司 用于免疫疗法的cd38组合物和方法
CA3238939A1 (en) 2021-12-08 2023-06-15 Gaurang Patel Mutant myocilin disease model and uses thereof
TW202332767A (zh) 2022-02-02 2023-08-16 美商雷傑納榮製藥公司 用於治療龐貝氏症之抗TfR:GAA及抗CD63:GAA插入
EP4514981A2 (en) 2022-04-29 2025-03-05 Regeneron Pharmaceuticals, Inc. Identification of tissue-specific extragenic safe harbors for gene therapy approaches
JP2025521154A (ja) 2022-05-31 2025-07-08 リジェネロン・ファーマシューティカルズ・インコーポレイテッド C9orf72反復伸長疾患のためのcrispr干渉療法
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
WO2023245113A1 (en) 2022-06-16 2023-12-21 Intellia Therapeutics, Inc. Methods and compositions for genetically modifying a cell
EP4547821A1 (en) 2022-06-29 2025-05-07 Intellia Therapeutics, Inc. Engineered t cells
JP2025528052A (ja) 2022-07-29 2025-08-26 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 脳及び筋肉へのトランスフェリン受容体(tfr)媒介送達のための組成物及び方法
CN120265314A (zh) 2022-09-28 2025-07-04 瑞泽恩制药公司 抗体抗性修饰受体以增强基于细胞的疗法
CN120693347A (zh) 2022-11-04 2025-09-23 瑞泽恩制药公司 钙电压门控通道辅助亚基γ1(CACNG1)结合蛋白和CACNG1介导的向骨骼肌的递送
EP4619438A2 (en) 2022-11-14 2025-09-24 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
WO2024137766A2 (en) 2022-12-21 2024-06-27 Intellia Therapeutics, Inc. Compositions and methods for proprotein convertase subtilisin kexin 9 (pcsk9) editing
TW202428878A (zh) 2022-12-23 2024-07-16 美商英特利亞醫療公司 用於基因體編輯之系統及方法
WO2024186890A1 (en) 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
TW202503051A (zh) 2023-03-07 2025-01-16 美商英特利亞醫療公司 用於免疫療法之cish組合物及方法
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
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2025006963A1 (en) 2023-06-30 2025-01-02 Regeneron Pharmaceuticals, Inc. Methods and compositions for increasing homology-directed repair
WO2025006782A2 (en) * 2023-06-30 2025-01-02 Chroma Medicine, Inc. Guide rna compositions
WO2025024486A2 (en) 2023-07-25 2025-01-30 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
WO2025024493A1 (en) 2023-07-25 2025-01-30 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
WO2025029654A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
WO2025029657A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
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)
WO2025038646A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Cd70 car-t compositions and methods for cell-based therapy
WO2025038648A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
WO2025038642A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying cd70
WO2025049524A1 (en) 2023-08-28 2025-03-06 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025101994A2 (en) 2023-11-10 2025-05-15 Intellia Therapeutics, Inc. Compositions, methods, and systems for genomic editing
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
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
WO2025137301A1 (en) 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Methods for rapid engineering of cells
WO2025137439A2 (en) 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Engineered t cells
WO2025184520A1 (en) 2024-02-29 2025-09-04 Intellia Therapeutics, Inc. Compositions and methods for angiopoietin like 3 (angptl3) editing
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
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT2784162E (pt) * 2012-12-12 2015-08-27 Broad Inst Inc Engenharia de sistemas, métodos e composições guia otimizadas para a manipulação de sequências
KR102874079B1 (ko) 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
EP3611268A1 (en) * 2013-08-22 2020-02-19 E. I. du Pont de Nemours and Company Plant genome modification using guide rna/cas endonuclease systems and methods of use
MX384887B (es) * 2014-06-23 2025-03-14 Regeneron Pharma Ensamblaje de adn mediado por nucleasa.
US20150376586A1 (en) 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
CN107250148B (zh) * 2014-12-03 2021-04-16 安捷伦科技有限公司 具有化学修饰的指导rna
ES2884838T3 (es) * 2015-04-06 2021-12-13 Univ Leland Stanford Junior ARN guía químicamente modificados para la regulación génica mediada por CRISPR/CAS
US11279928B2 (en) * 2015-06-29 2022-03-22 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
EP3159407A1 (en) * 2015-10-23 2017-04-26 Silence Therapeutics (London) Ltd Guide rnas, methods and uses
US11845933B2 (en) * 2016-02-03 2023-12-19 Massachusetts Institute Of Technology Structure-guided chemical modification of guide RNA and its applications
LT3436077T (lt) 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
SG11202002562QA (en) * 2017-09-29 2020-04-29 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing
WO2022170193A2 (en) * 2021-02-08 2022-08-11 Intellia Therapeutics, Inc. T-cell immunoglobulin and mucin domain 3 (tim3) compositions and methods for immunotherapy

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