DK3523430T3 - Manipulerede nukleinsyremålrettede nukleinsyrer - Google Patents
Manipulerede nukleinsyremålrettede nukleinsyrer Download PDFInfo
- Publication number
- DK3523430T3 DK3523430T3 DK17832598.1T DK17832598T DK3523430T3 DK 3523430 T3 DK3523430 T3 DK 3523430T3 DK 17832598 T DK17832598 T DK 17832598T DK 3523430 T3 DK3523430 T3 DK 3523430T3
- Authority
- DK
- Denmark
- Prior art keywords
- manipulated
- nucleic acid
- acid targeted
- nucleic acids
- acids
- Prior art date
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Classifications
-
- 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
- C12N15/111—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
- C12N15/10—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
- 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]
- C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Crystallography & Structural Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US15/371,188 US9816093B1 (en) | 2016-12-06 | 2016-12-06 | Engineered nucleic acid-targeting nucleic acids |
| US15/787,630 US10590415B2 (en) | 2016-12-06 | 2017-10-18 | Engineered nucleic acid-targeting nucleic acids |
| PCT/US2017/064771 WO2018106727A1 (en) | 2016-12-06 | 2017-12-05 | Engineered nuceic acid-targeting nucleic acids |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3523430T3 true DK3523430T3 (da) | 2021-07-26 |
Family
ID=60255607
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK17832598.1T DK3523430T3 (da) | 2016-12-06 | 2017-12-05 | Manipulerede nukleinsyremålrettede nukleinsyrer |
Country Status (4)
| Country | Link |
|---|---|
| US (3) | US9816093B1 (da) |
| EP (1) | EP3523430B1 (da) |
| DK (1) | DK3523430T3 (da) |
| WO (1) | WO2018106727A1 (da) |
Families Citing this family (56)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6261500B2 (ja) | 2011-07-22 | 2018-01-17 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ヌクレアーゼ切断特異性の評価および改善 |
| 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 |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| JP7109784B2 (ja) | 2015-10-23 | 2022-08-01 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 遺伝子編集のための進化したＣａｓ９蛋白質 |
| KR102547316B1 (ko) | 2016-08-03 | 2023-06-23 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| EP3526320A1 (en) | 2016-10-14 | 2019-08-21 | President and Fellows of Harvard College | Aav delivery of nucleobase editors |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| JP2020510038A (ja) | 2017-03-09 | 2020-04-02 | プレジデント アンド フェローズ オブ ハーバード カレッジ | がんワクチン |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | プレジデント アンド フェローズ オブ ハーバード カレッジ | シトシンからグアニンへの塩基編集因子 |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| 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 |
| 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 |
| WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| US20210093679A1 (en) | 2018-02-05 | 2021-04-01 | Novome Biotechnologies, Inc. | Engineered gut microbes and uses thereof |
| WO2019173248A1 (en) | 2018-03-07 | 2019-09-12 | Caribou Biosciences, Inc. | Engineered nucleic acid-targeting nucleic acids |
| KR20210045360A (ko) | 2018-05-16 | 2021-04-26 | 신테고 코포레이션 | 가이드 rna 설계 및 사용을 위한 방법 및 시스템 |
| EP3797160A1 (en) | 2018-05-23 | 2021-03-31 | The Broad Institute Inc. | Base editors and uses thereof |
| EP3823633A4 (en) | 2018-06-29 | 2023-05-03 | Editas Medicine, Inc. | SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| WO2020118110A1 (en) * | 2018-12-05 | 2020-06-11 | Fred Hutchinson Cancer Research Center | Reduced and minimal manipulation manufacturing of genetically-modified cells |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| US20220290120A1 (en) | 2019-02-25 | 2022-09-15 | Novome Biotechnologies, Inc. | Plasmids for gene editing |
| JP7657726B2 (ja) | 2019-03-19 | 2025-04-07 | ザ ブロード インスティテュート，インコーポレーテッド | 編集ヌクレオチド配列を編集するための方法および組成物 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| JP7345563B2 (ja) * | 2019-04-26 | 2023-09-15 | ツールゲン インコーポレイテッド | 標的特異的ｃｒｉｓｐｒ変異体 |
| AU2020311438A1 (en) * | 2019-07-11 | 2021-12-16 | Arbor Biotechnologies, Inc. | Novel CRISPR DNA targeting enzymes and systems |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| IL296024A (en) | 2020-03-04 | 2022-10-01 | Flagship Pioneering Innovations Vi Llc | Methods and compositions for modulating a genome |
| DE112021002672T5 (de) | 2020-05-08 | 2023-04-13 | President And Fellows Of Harvard College | Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz |
| IL308806A (en) | 2021-06-01 | 2024-01-01 | Arbor Biotechnologies Inc | Gene editing systems including nuclease crisper and their uses |
| JP2024533311A (ja) | 2021-09-08 | 2024-09-12 | フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー | ゲノムを調節するための方法及び組成物 |
| WO2023097268A2 (en) * | 2021-11-23 | 2023-06-01 | Mantra Bio, Inc. | Extracellular vesicles comprising non-naturally occurring modular rna hairpins and uses thereof |
| CN114685685B (zh) * | 2022-04-20 | 2023-11-07 | 上海科技大学 | 编辑rna的融合蛋白及其应用 |
| EP4286401A1 (en) * | 2022-06-03 | 2023-12-06 | Technische Universität Dresden | Use of an artificial protein or of a nucleic acid encoding the artificial protein for providing a functional ribonucleoprotein complex |
| EP4414452A1 (en) * | 2023-02-13 | 2024-08-14 | Universidad de Granada | Dual-guide rna composition for executing a single-guide rna crispr-associated system |
| WO2024173645A1 (en) | 2023-02-15 | 2024-08-22 | Arbor Biotechnologies, Inc. | Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript |
| AU2024299627A1 (en) | 2023-07-25 | 2026-01-22 | Flagship Pioneering Innovations Vii, Llc | Cas endonucleases and related methods |
| TW202519653A (zh) | 2023-07-25 | 2025-05-16 | 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 | Ｃａｓ內切酶及相關方法 |
| WO2025072331A1 (en) | 2023-09-26 | 2025-04-03 | Flagship Pioneering Innovations Vii, Llc | Cas nucleases and related methods |
| WO2025117877A2 (en) | 2023-12-01 | 2025-06-05 | Flagship Pioneering Innovations Vii, Llc | Cas nucleases and related methods |
| WO2025171210A1 (en) | 2024-02-09 | 2025-08-14 | Arbor Biotechnologies, Inc. | Compositions and methods for gene editing via homology-mediated end joining |
| WO2025194138A1 (en) | 2024-03-14 | 2025-09-18 | Tessera Therapeutics, Inc. | St1cas9 compositions and methods for modulating a genome |
| WO2026015829A2 (en) | 2024-07-12 | 2026-01-15 | Arbor Biotechnologies, Inc. | Small reverse transcriptases and gene editing systems comprising such |
| WO2026015832A2 (en) | 2024-07-12 | 2026-01-15 | Arbor Biotechnologies, Inc. | Reverse transcriptases and gene editing systems comprising such |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10087431B2 (en) | 2010-03-10 | 2018-10-02 | The Regents Of The University Of California | Methods of generating nucleic acid fragments |
| SG185481A1 (en) | 2010-05-10 | 2012-12-28 | Univ California | Endoribonuclease compositions and methods of use thereof |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| CN108285491B (zh) | 2012-02-29 | 2021-08-10 | 桑格摩生物科学股份有限公司 | 治疗亨廷顿氏病的方法和组合物 |
| US9637739B2 (en) | 2012-03-20 | 2017-05-02 | Vilnius University | RNA-directed DNA cleavage by the Cas9-crRNA complex |
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| WO2013188638A2 (en) | 2012-06-15 | 2013-12-19 | The Regents Of The University Of California | Endoribonucleases and methods of use thereof |
| HK1207111A1 (en) | 2012-08-03 | 2016-01-22 | 加利福尼亚大学董事会 | Methods and compositions for controlling gene expression by rna processing |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| CA2894684A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of improved crispr-cas systems, methods and enzyme compositions for sequence manipulation in eukaryotes |
| AU2013359262C1 (en) | 2012-12-12 | 2021-05-13 | Massachusetts Institute Of Technology | CRISPR-Cas component systems, methods and compositions for sequence manipulation |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| DK2931898T3 (da) | 2012-12-12 | 2016-06-20 | Massachusetts Inst Technology | Konstruktion og optimering af systemer, fremgangsmåder og sammensætninger til sekvensmanipulation med funktionelle domæner |
| CN105121648B (zh) | 2012-12-12 | 2021-05-07 | 布罗德研究所有限公司 | 用于序列操纵的系统、方法和优化的指导组合物的工程化 |
| MX374090B (es) | 2013-03-14 | 2025-03-05 | Caribou Biosciences Inc | Composiciones y métodos de ácidos nucleicos dirigidos al ácido nucleico. |
| CA3109801C (en) | 2013-08-22 | 2024-01-09 | Andrew Cigan | Plant genome modification using guide rna/cas endonuclease systems and methods of use |
| EP3760719A1 (en) | 2013-11-18 | 2021-01-06 | CRISPR Therapeutics AG | Crispr-cas system materials and methods |
| US20150376586A1 (en) | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
| EP3250691B9 (en) | 2015-01-28 | 2023-08-02 | Caribou Biosciences, Inc. | Crispr hybrid dna/rna polynucleotides and methods of use |
| EP3748004A1 (en) | 2015-04-01 | 2020-12-09 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy |
| US20160362667A1 (en) | 2015-06-10 | 2016-12-15 | Caribou Biosciences, Inc. | CRISPR-Cas Compositions and Methods |
-
2016
- 2016-12-06 US US15/371,188 patent/US9816093B1/en active Active
-
2017
- 2017-10-18 US US15/787,630 patent/US10590415B2/en active Active
- 2017-12-05 WO PCT/US2017/064771 patent/WO2018106727A1/en not_active Ceased
- 2017-12-05 DK DK17832598.1T patent/DK3523430T3/da active
- 2017-12-05 EP EP17832598.1A patent/EP3523430B1/en active Active
-
2020
- 2020-03-11 US US16/816,137 patent/US11001843B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| EP3523430B1 (en) | 2021-06-30 |
| US10590415B2 (en) | 2020-03-17 |
| US20180155720A1 (en) | 2018-06-07 |
| US11001843B2 (en) | 2021-05-11 |
| EP3523430A1 (en) | 2019-08-14 |
| US9816093B1 (en) | 2017-11-14 |
| US20200208155A1 (en) | 2020-07-02 |
| WO2018106727A1 (en) | 2018-06-14 |
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