JP2021526804A5 - - Google Patents

Info

Publication number
JP2021526804A5
JP2021526804A5 JP2020567951A JP2020567951A JP2021526804A5 JP 2021526804 A5 JP2021526804 A5 JP 2021526804A5 JP 2020567951 A JP2020567951 A JP 2020567951A JP 2020567951 A JP2020567951 A JP 2020567951A JP 2021526804 A5 JP2021526804 A5 JP 2021526804A5
Authority
JP
Japan
Prior art keywords
hairpin
grna
nucleotides
modification
modified nucleotide
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.)
Granted
Application number
JP2020567951A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021526804A (ja
JP7832761B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/036160 external-priority patent/WO2019237069A1/en
Publication of JP2021526804A publication Critical patent/JP2021526804A/ja
Publication of JP2021526804A5 publication Critical patent/JP2021526804A5/ja
Priority to JP2025052142A priority Critical patent/JP2025108457A/ja
Application granted granted Critical
Publication of JP7832761B2 publication Critical patent/JP7832761B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2020567951A 2018-06-08 2019-06-07 遺伝子編集のための修飾されたガイドrna Active JP7832761B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025052142A JP2025108457A (ja) 2018-06-08 2025-03-26 遺伝子編集のための修飾されたガイドrna

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862682838P 2018-06-08 2018-06-08
US201862682820P 2018-06-08 2018-06-08
US62/682,838 2018-06-08
US62/682,820 2018-06-08
PCT/US2019/036160 WO2019237069A1 (en) 2018-06-08 2019-06-07 Modified guide rnas for gene editing

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025052142A Division JP2025108457A (ja) 2018-06-08 2025-03-26 遺伝子編集のための修飾されたガイドrna

Publications (3)

Publication Number Publication Date
JP2021526804A JP2021526804A (ja) 2021-10-11
JP2021526804A5 true JP2021526804A5 (enExample) 2022-06-16
JP7832761B2 JP7832761B2 (ja) 2026-03-18

Family

ID=68770688

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020567951A Active JP7832761B2 (ja) 2018-06-08 2019-06-07 遺伝子編集のための修飾されたガイドrna
JP2025052142A Pending JP2025108457A (ja) 2018-06-08 2025-03-26 遺伝子編集のための修飾されたガイドrna

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025052142A Pending JP2025108457A (ja) 2018-06-08 2025-03-26 遺伝子編集のための修飾されたガイドrna

Country Status (16)

Country Link
US (1) US20210087568A1 (enExample)
EP (1) EP3802828A4 (enExample)
JP (2) JP7832761B2 (enExample)
KR (2) KR20260030838A (enExample)
CN (1) CN112567036A (enExample)
AU (2) AU2019282824C1 (enExample)
BR (1) BR112020024731A2 (enExample)
CA (1) CA3102950A1 (enExample)
CO (1) CO2021000051A2 (enExample)
IL (2) IL278822B1 (enExample)
MX (2) MX2020013293A (enExample)
PH (1) PH12020552100A1 (enExample)
SA (1) SA520420729B1 (enExample)
SG (1) SG11202011539VA (enExample)
TW (1) TW202016306A (enExample)
WO (1) WO2019237069A1 (enExample)

Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2019126483A (ru) 2017-01-23 2021-02-24 Ридженерон Фармасьютикалз, Инк. Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение
KR20190139869A (ko) 2017-04-11 2019-12-18 리제너론 파마슈티칼스 인코포레이티드 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CN110891420B (zh) 2017-07-31 2022-06-03 瑞泽恩制药公司 Cas转基因小鼠胚胎干细胞和小鼠及其应用
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
US20190098879A1 (en) 2017-09-29 2019-04-04 Regeneron Pharmaceuticals, Inc. Non-Human Animals 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
EP3592140A1 (en) 2018-03-19 2020-01-15 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
AU2019239971B2 (en) 2018-03-21 2025-09-11 Regeneron Pharmaceuticals, Inc. 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
AU2019360270B2 (en) 2018-10-18 2025-08-07 Intellia Therapeutics, Inc. Compositions and methods for expressing factor IX.
JP7631215B2 (ja) * 2019-03-28 2025-02-18 インテリア セラピューティクス,インコーポレイテッド Ttrガイドrnaと、rnaガイドdna結合剤をコードするポリヌクレオチドとを含む組成物および方法
US11891618B2 (en) 2019-06-04 2024-02-06 Regeneron Pharmaceuticals, Inc. Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use
MX2021015122A (es) 2019-06-07 2022-04-06 Regeneron Pharma Animales no humanos que comprenden un locus de albumina humanizado.
CN113906134B (zh) 2019-06-14 2025-06-24 瑞泽恩制药公司 Tau蛋白病模型
WO2021026318A2 (en) * 2019-08-06 2021-02-11 The Penn State Research Foundation Engineered crispr/cas9 systems for simultaneous long-term regulation of multiple targets
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
EP4438729A3 (en) * 2019-12-09 2025-03-26 Caribou Biosciences, Inc. Crispr abasic restricted nucleotides and crispr accuracy via analogs
EP4073249A1 (en) * 2019-12-11 2022-10-19 Intellia Therapeutics, Inc. Modified guide rnas for gene editing
BR112022015380A2 (pt) 2020-02-07 2022-09-27 Intellia Therapeutics Inc Composições e métodos para edição de gene de calicreína (klkb1)
MX2022012683A (es) * 2020-04-09 2023-01-11 Verve Therapeutics Inc Edicion de bases de pcsk9 y metodos de uso de la misma para el tratamiento de enfermedades.
CN115956120A (zh) * 2020-05-12 2023-04-11 马萨诸塞大学 用于crispr基因组编辑的修饰的指导rna
MX2023006878A (es) 2020-12-11 2023-07-31 Intellia Therapeutics Inc Composiciones y métodos para reducir el mhc de clase ii en una célula.
IL303506A (en) 2020-12-11 2023-08-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing involving deamination
WO2022140587A1 (en) 2020-12-23 2022-06-30 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying ciita in a cell
CR20230320A (es) 2020-12-23 2023-10-23 Intellia Therapeutics Inc Composiciones y métodos para reducir los hla-a en una célula
EP4288089A2 (en) 2021-02-08 2023-12-13 Intellia Therapeutics, Inc. T-cell immunoglobulin and mucin domain 3 (tim3) compositions and methods for immunotherapy
EP4288525A1 (en) 2021-02-08 2023-12-13 Intellia Therapeutics, Inc. Natural killer cell receptor 2b4 compositions and methods for immunotherapy
CN117042793A (zh) 2021-02-08 2023-11-10 因特利亚治疗公司 用于免疫疗法的淋巴细胞活化基因3(lag3)组合物和方法
AU2022227650A1 (en) * 2021-02-25 2023-10-12 Celyntra Therapeutics Sa Compositions and methods for targeting, editing, or modifying genes
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
JP2024540723A (ja) 2021-11-03 2024-11-01 インテリア セラピューティクス,インコーポレーテッド 免疫療法のためのcd38組成物及び方法
IL312507A (en) * 2021-11-03 2024-07-01 Intellia Therapeutics Inc Modified guide rnas for gene editing
KR20240117571A (ko) 2021-12-08 2024-08-01 리제너론 파마슈티칼스 인코포레이티드 돌연변이 마이오실린 질환 모델 및 이의 용도
WO2023185697A2 (en) * 2022-03-29 2023-10-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
CN114574598B (zh) * 2022-04-02 2023-08-08 中国农业科学院北京畜牧兽医研究所 鉴定或辅助鉴定猪5/6肋眼肌面积的方法
KR20250037788A (ko) * 2022-06-15 2025-03-18 빔 테라퓨틱스, 인크. 보체 활성화를 감소시키기 위한 조성물 및 방법
CN119384498A (zh) 2022-06-16 2025-01-28 因特利亚治疗公司 用于对细胞进行遗传修饰的方法和组合物
AU2023293131A1 (en) 2022-06-16 2024-12-12 Intellia Therapeutics, Inc. Compositions and methods for reducing mhc class i in a cell
WO2024006955A1 (en) 2022-06-29 2024-01-04 Intellia Therapeutics, Inc. Engineered t cells
WO2024003810A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Guide rna with chemical modifications
KR20250124819A (ko) 2022-12-21 2025-08-20 인텔리아 테라퓨틱스, 인크. 프로단백질 전환효소 서브틸리신 켁신 9(pcsk9) 편집을 위한 조성물 및 방법
CN120418427A (zh) 2022-12-23 2025-08-01 因特利亚治疗公司 用于基因组编辑的系统和方法
TW202436622A (zh) 2023-03-06 2024-09-16 美商英特利亞醫療公司 用於b型肝炎病毒(hbv)基因體編輯之組合物及方法
WO2024186971A1 (en) 2023-03-07 2024-09-12 Intellia Therapeutics, Inc. Cish compositions and methods for immunotherapy
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
CN119265190A (zh) * 2023-07-06 2025-01-07 上海交通大学 一种靶向TTR的saCas9 sgRNA及其修饰方式
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
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)
WO2025059328A1 (en) * 2023-09-14 2025-03-20 Empirico Inc. Modified oligonucleotides
WO2025101994A2 (en) 2023-11-10 2025-05-15 Intellia Therapeutics, Inc. Compositions, methods, and systems for genomic editing
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
WO2025137439A2 (en) 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Engineered t cells
WO2025137301A1 (en) 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Methods for rapid engineering of cells
WO2025184520A1 (en) 2024-02-29 2025-09-04 Intellia Therapeutics, Inc. Compositions and methods for angiopoietin like 3 (angptl3) editing
CN117925623B (zh) * 2024-03-20 2024-06-07 北京引正基因科技有限公司 用于hao1基因编辑和治疗ph1的组合物
WO2025240946A1 (en) 2024-05-17 2025-11-20 Intellia Therapeutics, Inc. Lipid nanoparticles and lipid nanoparticle compositions
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy
WO2026006390A2 (en) * 2024-06-26 2026-01-02 Intellia Therapeutics, Inc. Modified guide rnas for genome editing

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150376586A1 (en) 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
US10513711B2 (en) * 2014-08-13 2019-12-24 Dupont Us Holding, Llc Genetic targeting in non-conventional yeast using an RNA-guided endonuclease
CA2969619A1 (en) * 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
CA2970683A1 (en) * 2014-12-18 2016-06-23 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
KR102888521B1 (ko) * 2015-04-06 2025-11-19 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
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
WO2017136794A1 (en) * 2016-02-03 2017-08-10 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
WO2019067910A1 (en) * 2017-09-29 2019-04-04 Intellia Therapeutics, Inc. POLYNUCLEOTIDES, COMPOSITIONS AND METHODS FOR GENOMIC EDITION

Similar Documents

Publication Publication Date Title
JP2021526804A5 (enExample)
JP2023506482A5 (enExample)
JP7625555B2 (ja) 修飾されたガイドrna
US12391722B2 (en) Stabilized nucleic acids encoding messenger ribonucleic acid (mRNA)
JP6959293B2 (ja) B型肝炎ウイルスの遺伝子発現を阻害するための組成物および方法
JP5805088B2 (ja) 遺伝子発現を阻害する組成物およびその使用
JP7244922B2 (ja) 化学修飾された一本鎖rna編集オリゴヌクレオチド
JP2024541962A5 (enExample)
CN114502204A (zh) 具有降低的免疫刺激性质的rna组合和组合物
EP3159407A1 (en) Guide rnas, methods and uses
JP2018519811A5 (enExample)
JP2021519071A (ja) シュードウリジン化のための核酸分子
JP2023519274A5 (enExample)
JP2021518102A5 (enExample)
JP2012050447A (ja) 染色体dnaに標的化されるオリゴマーによる遺伝子発現の調節
JP2009535039A5 (enExample)
JP2003505080A (ja) ヒトeg5の発現を阻害するためのオリゴヌクレオチド
JP2024529425A5 (enExample)
KR20010042848A (ko) 인슐린유사 성장인자 ⅱ의 안티센스 올리고뉴클레오타이드서열 및 이를 이용한 세포성장 조절방법
IL159759A (en) History of oligoribonucleotide for deliberate inhibition of gene expression, medicinal preparations containing them, methods for their preparation and use
JP2024516575A5 (enExample)
WO2020154594A2 (en) Compositions and methods for treatment of human papillomavirus
JP2025514327A (ja) Crisprガイドrnaのための保護オリゴヌクレオチド
HK40061440A (zh) 用於抑制乙型肝炎病毒的基因表达的组合物和方法
HK40116631A (zh) 修饰的基於crispr的基因编辑系统及其使用方法