IL293569A - A modified rnas guide to gene editing - Google Patents
A modified rnas guide to gene editingInfo
- Publication number
- IL293569A IL293569A IL293569A IL29356922A IL293569A IL 293569 A IL293569 A IL 293569A IL 293569 A IL293569 A IL 293569A IL 29356922 A IL29356922 A IL 29356922A IL 293569 A IL293569 A IL 293569A
- Authority
- IL
- Israel
- Prior art keywords
- grna
- modification
- nucleotides
- region
- substituted
- Prior art date
Links
Classifications
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- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
- 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
- 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
- C12N2310/34—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
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
-
- 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
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Crystallography & Structural Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962946905P | 2019-12-11 | 2019-12-11 | |
| PCT/US2020/064250 WO2021119275A1 (en) | 2019-12-11 | 2020-12-10 | Modified guide rnas for gene editing |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL293569A true IL293569A (en) | 2022-08-01 |
Family
ID=74181290
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL293569A IL293569A (en) | 2019-12-11 | 2020-12-10 | A modified rnas guide to gene editing |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20220372483A1 (https=) |
| EP (1) | EP4073249A1 (https=) |
| JP (2) | JP2023506482A (https=) |
| KR (1) | KR20220126725A (https=) |
| CN (1) | CN115176001A (https=) |
| AU (1) | AU2020401206A1 (https=) |
| BR (1) | BR112022011214A2 (https=) |
| CA (1) | CA3164192A1 (https=) |
| CO (1) | CO2022009562A2 (https=) |
| IL (1) | IL293569A (https=) |
| MX (1) | MX2022006950A (https=) |
| TW (1) | TW202136509A (https=) |
| WO (1) | WO2021119275A1 (https=) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20240038705A (ko) * | 2021-06-22 | 2024-03-25 | 인텔리아 테라퓨틱스, 인크. | 간 유전자의 생체 내 편집 방법 |
| IL312507A (en) * | 2021-11-03 | 2024-07-01 | Intellia Therapeutics Inc | Modified guide rnas for gene editing |
| 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 |
| 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 |
| 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 |
| 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) |
| WO2025038642A1 (en) | 2023-08-14 | 2025-02-20 | Intellia Therapeutics, Inc. | Compositions and methods for genetically modifying cd70 |
| WO2025038646A1 (en) | 2023-08-14 | 2025-02-20 | Intellia Therapeutics, Inc. | Cd70 car-t compositions and methods for cell-based therapy |
| 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 |
| 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 |
| WO2025255308A1 (en) | 2024-06-07 | 2025-12-11 | Intellia Therapeutics, Inc. | Cd8 co-receptor chimeric polypeptides in tcr cell therapy |
| WO2025265017A1 (en) | 2024-06-20 | 2025-12-26 | Regeneron Pharmaceuticals, Inc. | Ass1 gene insertion for the treatment of citrullinemia type i |
| WO2026006390A2 (en) * | 2024-06-26 | 2026-01-02 | Intellia Therapeutics, Inc. | Modified guide rnas for genome editing |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| JP4236812B2 (ja) | 1997-09-12 | 2009-03-11 | エクシコン エ／エス | オリゴヌクレオチド類似体 |
| WO2001075436A1 (en) | 2000-04-03 | 2001-10-11 | Board Of Trustees Of The Leland Stanford Junior University | A method for protein structure alignment |
| US7774185B2 (en) | 2004-09-14 | 2010-08-10 | International Business Machines Corporation | Protein structure alignment using cellular automata |
| CN112301024A (zh) | 2013-03-15 | 2021-02-02 | 通用医疗公司 | 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性 |
| WO2014204724A1 (en) * | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| JP2017503514A (ja) * | 2014-01-24 | 2017-02-02 | ノースカロライナ ステート ユニバーシティーＮｏｒｔｈ Ｃａｒｏｌｉｎａ Ｓｔａｔｅ Ｕｎｉｖｅｒｓｉｔｙ | Ｃａｓ９ターゲッティングをガイドする配列に関する方法および組成物 |
| US20150376586A1 (en) * | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
| WO2015200555A2 (en) * | 2014-06-25 | 2015-12-30 | Caribou Biosciences, Inc. | Rna modification to engineer cas9 activity |
| CN106794141B (zh) | 2014-07-16 | 2021-05-28 | 诺华股份有限公司 | 将核酸包封在脂质纳米粒主体中的方法 |
| CA2969619A1 (en) * | 2014-12-03 | 2016-06-09 | Agilent Technologies, Inc. | Guide rna with chemical modifications |
| 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 |
| SG10202106058WA (en) * | 2016-12-08 | 2021-07-29 | Intellia Therapeutics Inc | Modified guide rnas |
| EP3688161A1 (en) * | 2017-09-29 | 2020-08-05 | Intellia Therapeutics, Inc. | Compositions and methods for ttr gene editing and treating attr amyloidosis |
| WO2019067910A1 (en) | 2017-09-29 | 2019-04-04 | Intellia Therapeutics, Inc. | POLYNUCLEOTIDES, COMPOSITIONS AND METHODS FOR GENOMIC EDITION |
| US20230140670A1 (en) * | 2017-09-29 | 2023-05-04 | Intellia Therapeutics, Inc. | Formulations |
| AU2019282824C1 (en) * | 2018-06-08 | 2026-04-23 | Intellia Therapeutics, Inc. | Modified guide RNAS for gene editing |
-
2020
- 2020-12-10 EP EP20839432.0A patent/EP4073249A1/en active Pending
- 2020-12-10 BR BR112022011214A patent/BR112022011214A2/pt unknown
- 2020-12-10 WO PCT/US2020/064250 patent/WO2021119275A1/en not_active Ceased
- 2020-12-10 MX MX2022006950A patent/MX2022006950A/es unknown
- 2020-12-10 IL IL293569A patent/IL293569A/en unknown
- 2020-12-10 KR KR1020227023444A patent/KR20220126725A/ko active Pending
- 2020-12-10 TW TW109143632A patent/TW202136509A/zh unknown
- 2020-12-10 CN CN202080094797.XA patent/CN115176001A/zh active Pending
- 2020-12-10 AU AU2020401206A patent/AU2020401206A1/en active Pending
- 2020-12-10 CA CA3164192A patent/CA3164192A1/en active Pending
- 2020-12-10 JP JP2022535572A patent/JP2023506482A/ja active Pending
-
2022
- 2022-06-09 US US17/836,265 patent/US20220372483A1/en active Pending
- 2022-07-07 CO CONC2022/0009562A patent/CO2022009562A2/es unknown
-
2025
- 2025-12-04 JP JP2025229544A patent/JP2026048746A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CN115176001A (zh) | 2022-10-11 |
| US20220372483A1 (en) | 2022-11-24 |
| TW202136509A (zh) | 2021-10-01 |
| BR112022011214A2 (pt) | 2022-08-23 |
| AU2020401206A1 (en) | 2022-07-14 |
| CO2022009562A2 (es) | 2022-08-30 |
| JP2023506482A (ja) | 2023-02-16 |
| WO2021119275A1 (en) | 2021-06-17 |
| MX2022006950A (es) | 2022-11-07 |
| CA3164192A1 (en) | 2021-06-17 |
| EP4073249A1 (en) | 2022-10-19 |
| KR20220126725A (ko) | 2022-09-16 |
| JP2026048746A (ja) | 2026-03-17 |
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