JP7832761B2 - 遺伝子編集のための修飾されたガイドｒｎａ - Google Patents

遺伝子編集のための修飾されたガイドｒｎａ

Info

Publication number
JP7832761B2
JP7832761B2 JP2020567951A JP2020567951A JP7832761B2 JP 7832761 B2 JP7832761 B2 JP 7832761B2 JP 2020567951 A JP2020567951 A JP 2020567951A JP 2020567951 A JP2020567951 A JP 2020567951A JP 7832761 B2 JP7832761 B2 JP 7832761B2
Authority
JP
Japan
Prior art keywords
nucleotides
modification
grna
nucleotide
region
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
JP2020567951A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2019237069A5 (https=
JP2021526804A (ja
JP2021526804A5 (https=
Inventor
マディソン ローデン スミス，エイミー
アレクサンダー，セス
ブラウン，ダンカン
ブラウン，ロバート
ランバ，アドヒラ
レスカルボ―，レベッカ
マイケル レスカルボ―，レイナルド
パルマー，ルビナ
ロイ，マシュー
サレルノ，ペイジ
リン サイツァー，ジェシカ
Original Assignee
インテリア セラピューティクス，インコーポレイテッド
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 インテリア セラピューティクス，インコーポレイテッド filed Critical インテリア セラピューティクス，インコーポレイテッド
Publication of JP2021526804A publication Critical patent/JP2021526804A/ja
Publication of JPWO2019237069A5 publication Critical patent/JPWO2019237069A5/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

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/1137—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 against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Mycology (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
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
US62/682,820 2018-06-08
US62/682,838 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 (4)

Publication Number Publication Date
JP2021526804A JP2021526804A (ja) 2021-10-11
JPWO2019237069A5 JPWO2019237069A5 (https=) 2022-06-16
JP2021526804A5 JP2021526804A5 (https=) 2022-06-16
JP7832761B2 true 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 (https=)
EP (1) EP3802828A4 (https=)
JP (2) JP7832761B2 (https=)
KR (2) KR102919364B1 (https=)
CN (1) CN112567036A (https=)
AU (2) AU2019282824C1 (https=)
BR (1) BR112020024731A2 (https=)
CA (1) CA3102950A1 (https=)
CO (1) CO2021000051A2 (https=)
IL (2) IL278822B1 (https=)
MX (2) MX2020013293A (https=)
PH (1) PH12020552100A1 (https=)
SA (1) SA520420729B1 (https=)
SG (1) SG11202011539VA (https=)
TW (1) TW202016306A (https=)
WO (1) WO2019237069A1 (https=)

Families Citing this family (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL314915B1 (en) 2017-01-23 2026-02-01 Regeneron Pharma Variants of 17-beta-hydroxysteroid dehydrogenase and their uses
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
WO2019028032A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. EMBRYONIC STEM CELLS OF TRANSGENIC MOUSE CASES AND MICE AND USES THEREOF
CN111182790A (zh) 2017-07-31 2020-05-19 瑞泽恩制药公司 Crispr报告体非人类动物及其用途
MX394999B (es) 2017-09-29 2025-03-24 Regeneron Pharma Animales no humanos que comprenden un locus ttr humanizado y metodos de uso
US10961583B2 (en) 2017-10-11 2021-03-30 Regeneron Phramaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
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
JP7507093B2 (ja) 2018-03-21 2024-06-27 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 １７β－水酸化ステロイド脱水素酵素１３型（ＨＳＤ１７Ｂ１３）ｉＲＮＡ組成物およびその使用方法
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
JP7578590B2 (ja) 2018-10-18 2024-11-06 インテリア セラピューティクス，インコーポレーテッド 第ｉｘ因子を発現するための組成物及び方法
EP3946598A1 (en) * 2019-03-28 2022-02-09 Intellia Therapeutics, Inc. Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent
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
US11622547B2 (en) 2019-06-07 2023-04-11 Regeneran Pharmaceuticals, Inc. Genetically modified mouse that expresses human albumin
US11845957B2 (en) 2019-06-14 2023-12-19 Regeneron Pharmaceuticals, Inc. Models of tauopathy
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
MX2022006950A (es) * 2019-12-11 2022-11-07 Intellia Therapeutics Inc Arn guía modificados para edición de genes.
JP2023512758A (ja) 2020-02-07 2023-03-29 インテリア セラピューティクス，インコーポレイテッド カリクレイン（ｋｌｋｂ１）遺伝子編集のための組成物および方法
GB2632565B (en) * 2020-04-09 2025-06-04 Verve Therapeutics Inc Base editing of PCSK9 and methods of using same for treatment of disease
WO2021231606A2 (en) * 2020-05-12 2021-11-18 University Of Massachusetts Modified guide rnas for crispr genome editing
CA3204997A1 (en) 2020-12-11 2022-06-16 Intellia Therapeutics, Inc. Compositions and methods for reducing mhc class ii in a cell
CR20230305A (es) 2020-12-11 2023-11-10 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación
AU2021409732A1 (en) 2020-12-23 2023-07-20 Intellia Therapeutics, Inc. Compositions and methods for reducing hla-a in a cell
TW202242101A (zh) 2020-12-23 2022-11-01 美商英特利亞醫療公司 用於細胞中基因修飾ciita之組合物及方法
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
JP2024506016A (ja) 2021-02-08 2024-02-08 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ｔ細胞免疫グロブリン及びムチンドメイン３（ｔｉｍ３）組成物及び方法
WO2022170194A2 (en) 2021-02-08 2022-08-11 Intellia Therapeutics, Inc. Lymphocyte activation gene 3 (lag3) compositions and methods for immunotherapy
EP4298212A4 (en) * 2021-02-25 2026-04-15 Celyntra Therapeutics Sa Compositions and methods for targeting, editing, or modifying genes
EP4392060A1 (en) 2021-08-24 2024-07-03 Intellia Therapeutics, Inc. Programmed cell death protein 1 (pd1) compositions and methods for cell-based therapy
EP4426338A2 (en) 2021-11-03 2024-09-11 Intellia Therapeutics, Inc. Cd38 compositions and methods for immunotherapy
WO2023081687A1 (en) * 2021-11-03 2023-05-11 Intellia Therapeutics, Inc. Modified guide rnas for gene editing
CN118632622A (zh) 2021-12-08 2024-09-10 瑞泽恩制药公司 突变型肌纤蛋白疾病模型及其用途
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
CN119923466A (zh) * 2022-06-15 2025-05-02 比姆医疗股份有限公司 用于减少补体激活的组合物和方法
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
EP4540389A2 (en) 2022-06-16 2025-04-23 Intellia Therapeutics, Inc. Compositions and methods for reducing mhc class i in a cell
CN119585419A (zh) 2022-06-29 2025-03-07 因特利亚治疗公司 工程化t细胞
TW202417017A (zh) * 2022-06-30 2024-05-01 香港商正基基因科技有限公司 具有化學修飾的導引rna
WO2024137766A2 (en) 2022-12-21 2024-06-27 Intellia Therapeutics, Inc. Compositions and methods for proprotein convertase subtilisin kexin 9 (pcsk9) editing
WO2024138115A1 (en) 2022-12-23 2024-06-27 Intellia Theraperutics, Inc. Systems and methods for genomic editing
WO2024186890A1 (en) 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
EP4677067A1 (en) 2023-03-07 2026-01-14 Intellia Therapeutics, Inc. Cish compositions and methods for immunotherapy
IL324634A (en) 2023-05-15 2026-01-01 Nchroma Bio Inc Compositions and methods for epigenetic regulation of hbv gene expression
WO2025007951A1 (zh) * 2023-07-06 2025-01-09 上海交通大学 一种靶向ttr的sacas9 sgrna及其修饰方式
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)
TW202515994A (zh) 2023-08-14 2025-04-16 美商英特利亞醫療公司 用於對ｃｄ７０進行基因修飾之組合物及方法
TW202521564A (zh) 2023-08-14 2025-06-01 美商英特利亞醫療公司 用於基於細胞之療法的ｃｄ７０ ｃａｒ－ｔ組合物及方法
WO2025059328A1 (en) * 2023-09-14 2025-03-20 Empirico Inc. Modified oligonucleotides
TW202540417A (zh) 2023-11-10 2025-10-16 美商英特利亞醫療公司 用於基因體編輯之組合物、方法及系統
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017537626A (ja) 2014-12-03 2017-12-21 アジレント・テクノロジーズ・インクＡｇｉｌｅｎｔ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． 化学修飾を有するガイドｒｎａ
JP2017538427A (ja) 2014-12-18 2017-12-28 インテグレイテッド ディーエヌエイ テクノロジーズ インコーポレイテッド Ｃｒｉｓｐｒ系組成物及び使用方法

Family Cites Families (8)

* 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
EP3280803B1 (en) * 2015-04-06 2021-05-26 The Board of Trustees of the Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
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
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
AR113154A1 (es) * 2017-09-29 2020-01-29 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para edición del genoma

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017537626A (ja) 2014-12-03 2017-12-21 アジレント・テクノロジーズ・インクＡｇｉｌｅｎｔ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． 化学修飾を有するガイドｒｎａ
JP2017538427A (ja) 2014-12-18 2017-12-28 インテグレイテッド ディーエヌエイ テクノロジーズ インコーポレイテッド Ｃｒｉｓｐｒ系組成物及び使用方法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Nishimasu, H. et al.，"Crystal structure of Cas9 in complex with guide RNA and target DNA"，Cell，2014年，Vol. 156，pp. 935-949
Yin, H. et al.，"Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing"，Nat. Biotechnol.，2017年，Vol. 35，pp. 1179-1187

Also Published As

Publication number Publication date
KR20210029772A (ko) 2021-03-16
KR102919364B1 (ko) 2026-01-29
AU2019282824C1 (en) 2026-04-23
JP2025108457A (ja) 2025-07-23
MX2025001112A (es) 2025-03-07
KR20260030838A (ko) 2026-03-06
PH12020552100A1 (en) 2021-08-02
CO2021000051A2 (es) 2021-01-18
AU2019282824B2 (en) 2025-10-23
SG11202011539VA (en) 2020-12-30
AU2019282824A1 (en) 2021-01-07
MX2020013293A (es) 2021-05-12
SA520420729B1 (ar) 2024-03-17
IL278822A (en) 2021-01-31
IL278822B1 (en) 2026-04-01
WO2019237069A1 (en) 2019-12-12
AU2026200415A1 (en) 2026-02-19
EP3802828A1 (en) 2021-04-14
CA3102950A1 (en) 2019-12-12
US20210087568A1 (en) 2021-03-25
BR112020024731A2 (pt) 2021-03-23
TW202016306A (zh) 2020-05-01
CN112567036A (zh) 2021-03-26
IL326752A (en) 2026-04-01
EP3802828A4 (en) 2022-10-26
JP2021526804A (ja) 2021-10-11

Similar Documents

Publication Publication Date Title
JP7832761B2 (ja) 遺伝子編集のための修飾されたガイドｒｎａ
US20220372483A1 (en) Modified Guide RNAs for Gene Editing
JP2025165976A (ja) オリゴヌクレオチド組成物及びその使用方法
JP2022126736A (ja) 修飾されたガイドｒｎａ
US20240026358A1 (en) Oligonucleotide compositions and methods thereof
US20230392137A1 (en) Oligonucleotide compositions and methods thereof
DE69736840T2 (de) Synthese von nukleosiden und polynukleotiden
CA3222745A1 (en) Modified guide rnas comprising an internal linker for gene editing
TW202334419A (zh) 用於基因編輯之經修飾引導rna
EP3728591B1 (en) Gapmer oligonucleotides comprising a phosphorodithioate internucleoside linkage
CN113677793A (zh) 稳定的crispr复合物
CN121079412A (zh) 经修饰的指导rna
JP2025536808A (ja) 遺伝性ｈｆｅヘモクロマトーシスの治療のためのアンチセンスオリゴヌクレオチド
Eschgfäller et al. Synthesis and biodistribution of a short nonionic oligonucleotide analogue in mouse with a potential to mimic peptides
HK40078499A (en) Modified guide rnas for gene editing
Montulet Optimization of the Chemical Modification and Conjugation of Exon Skipping Antisense Oligonucleotides for the Gene Therapy of Duchenne Muscular Dystrophy
HK40048090A (en) Modified guide rnas for gene editing
HK40060724A (en) Stabilized crispr complexes
JP2025525564A (ja) オリゴヌクレオチドの編集
CN121399260A (zh) 用于治疗神经退行性疾病的反义寡核苷酸
WO2023147479A1 (en) Conjugated crispr-cas complexes
HK40014268A (en) Modified guide rnas

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220607

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220607

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230613

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230810

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231213

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240206

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240318

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240805

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20241126

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250326

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250729

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20251009

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260128

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: 20260210

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20260306

R150 Certificate of patent or registration of utility model

Ref document number: 7832761

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150