CA3201258A1 - Engineered class 2 type v crispr systems - Google Patents

Engineered class 2 type v crispr systems

Info

Publication number
CA3201258A1
CA3201258A1 CA3201258A CA3201258A CA3201258A1 CA 3201258 A1 CA3201258 A1 CA 3201258A1 CA 3201258 A CA3201258 A CA 3201258A CA 3201258 A CA3201258 A CA 3201258A CA 3201258 A1 CA3201258 A1 CA 3201258A1
Authority
CA
Canada
Prior art keywords
seq
grna
sequence
fold
protein
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.)
Pending
Application number
CA3201258A
Other languages
English (en)
French (fr)
Inventor
Sean Higgins
Isabel COLIN
Sarah DENNY
Brett T. STAAHL
Benjamin OAKES
Angus SIDORE
Suraj MAKHIJA
Gayathri VIJAYAKUMAR
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Scribe Therapeutics Inc
Original Assignee
Scribe Therapeutics Inc
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 Scribe Therapeutics Inc filed Critical Scribe Therapeutics Inc
Publication of CA3201258A1 publication Critical patent/CA3201258A1/en
Pending legal-status Critical Current

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    • 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
    • C12N15/113—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
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (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)
  • Virology (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CA3201258A 2020-12-03 2021-12-02 Engineered class 2 type v crispr systems Pending CA3201258A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063121196P 2020-12-03 2020-12-03
US63/121,196 2020-12-03
US202163162346P 2021-03-17 2021-03-17
US63/162,346 2021-03-17
US202163208855P 2021-06-09 2021-06-09
US63/208,855 2021-06-09
PCT/US2021/061673 WO2022120095A1 (en) 2020-12-03 2021-12-02 Engineered class 2 type v crispr systems

Publications (1)

Publication Number Publication Date
CA3201258A1 true CA3201258A1 (en) 2022-06-09

Family

ID=80050633

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3201258A Pending CA3201258A1 (en) 2020-12-03 2021-12-02 Engineered class 2 type v crispr systems

Country Status (11)

Country Link
US (4) US20240026385A1 (https=)
EP (1) EP4256054A1 (https=)
JP (1) JP2023552374A (https=)
KR (1) KR20230128289A (https=)
AU (1) AU2021392719B2 (https=)
CA (1) CA3201258A1 (https=)
CL (2) CL2023001594A1 (https=)
GB (1) GB2616584B (https=)
MX (1) MX2023006566A (https=)
PE (1) PE20231178A1 (https=)
WO (1) WO2022120095A1 (https=)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3980533A1 (en) 2019-06-07 2022-04-13 Scribe Therapeutics Inc. Engineered casx systems
WO2021050601A1 (en) 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy
WO2021113763A1 (en) 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
KR20230128289A (ko) 2020-12-03 2023-09-04 스크라이브 테라퓨틱스 인크. 조작된 클래스 2 유형 v crispr 시스템
WO2022261149A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
GB2625500B (en) 2021-09-21 2026-04-01 Scribe Therapeutics Inc Engineered CasX repressor systems
WO2023235818A2 (en) 2022-06-02 2023-12-07 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
WO2023235888A2 (en) 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
TW202413643A (zh) 2022-06-07 2024-04-01 美商斯奎柏治療公司 用於靶向pcsk9的組合物及方法
WO2023240157A2 (en) 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of dmd
US20250361525A1 (en) 2022-06-08 2025-11-27 Scribe Therapeutics Inc. Aav vectors for gene editing
CN116676291B (zh) * 2022-08-22 2024-02-27 华中农业大学 核酸内切酶Genie scissor及其介导的基因编辑系统
WO2024206565A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Repressor fusion protein systems
PE20252746A1 (es) 2023-03-29 2025-12-05 Scribe Therapeutics Inc Composiciones y metodos para el direccionamiento a pcsk9
WO2024206676A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of lpa
WO2024206620A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Messenger rna encoding casx
JP7560818B1 (ja) 2023-08-25 2024-10-03 株式会社キュライオ エンジニアリングされたタンパク質
AU2024332871A1 (en) * 2023-08-25 2026-03-12 Jichi Medical University Engineered protein
WO2025072293A1 (en) 2023-09-27 2025-04-03 Scribe Therapeutics Inc. Optimized mrnas encoding casx proteins
WO2025240940A1 (en) 2024-05-17 2025-11-20 Scribe Therapeutics Inc. Compositions and methods for the targeting of apolipoprotein c3

Family Cites Families (78)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5208036A (en) 1985-01-07 1993-05-04 Syntex (U.S.A.) Inc. N-(ω, (ω-1)-dialkyloxy)- and N-(ω, (ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5223409A (en) 1988-09-02 1993-06-29 Protein Engineering Corp. Directed evolution of novel binding proteins
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5412087A (en) 1992-04-24 1995-05-02 Affymax Technologies N.V. Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces
EP1179340A3 (en) 1994-09-30 2003-05-07 INEX Pharmaceutical Corp. Compositions for the introduction of polyanionic materials into cells
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5695937A (en) 1995-09-12 1997-12-09 The Johns Hopkins University School Of Medicine Method for serial analysis of gene expression
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
WO2007086881A2 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
CA2742969A1 (en) 2008-11-07 2010-05-14 Fabrus Llc Anti-dll4 antibodies and uses thereof
WO2010075303A1 (en) 2008-12-23 2010-07-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same
US20110071208A1 (en) 2009-06-05 2011-03-24 Protiva Biotherapeutics, Inc. Lipid encapsulated dicer-substrate interfering rna
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
CA2769670C (en) 2009-07-31 2018-10-02 Ethris Gmbh Rna with a combination of unmodified and modified nucleotides for protein expression
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
HUE060907T2 (hu) 2014-06-25 2023-04-28 Acuitas Therapeutics Inc Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére
CA2969619A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
JP2018509387A (ja) 2015-01-21 2018-04-05 フェーズアールエックス インコーポレイテッド 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016279077A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
PT3313829T (pt) 2015-06-29 2024-07-08 Acuitas Therapeutics Inc Formulações de lípidos e de nanopartículas lipídicas para a administração de ácidos nucleicos
US20190048340A1 (en) 2015-09-24 2019-02-14 Crispr Therapeutics Ag Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
CN116814590A (zh) 2015-10-22 2023-09-29 布罗德研究所有限公司 Vi-b型crispr酶和系统
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
EP3390624A4 (en) 2015-12-18 2019-07-10 The Regents of The University of California Modified site-directed modifying polypeptides and methods of use thereof
US9896696B2 (en) 2016-02-15 2018-02-20 Benson Hill Biosystems, Inc. Compositions and methods for modifying genomes
WO2017212007A1 (en) 2016-06-09 2017-12-14 Curevac Ag Cationic carriers for nucleic acid delivery
EP3468612A1 (en) 2016-06-09 2019-04-17 CureVac AG Hybrid carriers for nucleic acid cargo
AU2017280353B2 (en) 2016-06-24 2021-11-11 Inscripta, Inc. Methods for generating barcoded combinatorial libraries
EP3500967A1 (en) 2016-08-17 2019-06-26 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
MX2019003674A (es) 2016-09-30 2021-01-08 Univ California Enzimas modificadoras de ácido nucleico guiadas por arn y métodos de uso de estas.
US9982267B2 (en) 2016-10-12 2018-05-29 Feldan Bio Inc. Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same
ES2928176T3 (es) 2017-01-10 2022-11-16 Christiana Care Gene Editing Inst Llc Métodos para mutagénesis dirigida al sitio in vitro mediante el uso de tecnologías de edición de genes
US20190367924A1 (en) 2017-02-17 2019-12-05 Temple University - Of The Commonwealth System Of Higher Education Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
SG11201906297QA (en) 2017-03-24 2019-10-30 Curevac Ag Nucleic acids encoding crispr-associated proteins and uses thereof
WO2018195555A1 (en) 2017-04-21 2018-10-25 The Board Of Trustees Of The Leland Stanford Junior University Crispr/cas 9-mediated integration of polynucleotides by sequential homologous recombination of aav donor vectors
US11578334B2 (en) 2017-10-25 2023-02-14 Monsanto Technology Llc Targeted endonuclease activity of the RNA-guided endonuclease CasX in eukaryotes
US10253365B1 (en) 2017-11-22 2019-04-09 The Regents Of The University Of California Type V CRISPR/Cas effector proteins for cleaving ssDNAs and detecting target DNAs
US20200224160A1 (en) 2018-02-27 2020-07-16 Sorrento Therapeutics, Inc. Process for dna integration using rna-guided endonucleases
WO2019168950A1 (en) 2018-02-27 2019-09-06 The University Of North Carolina At Chapel Hill Methods and compositions for treating angelman syndrome
WO2019213273A1 (en) 2018-05-01 2019-11-07 The Children's Medical Center Corporation Enhanced bcl11a rnp / crispr delivery & editing using a 3xnls-cas9
EP3818167A4 (en) 2018-07-05 2022-04-13 The Regents of The University of California COMPOSITIONS AND METHODS FOR DELIVERING RNA TO A CELL
US20210284981A1 (en) 2018-07-24 2021-09-16 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
US12275964B2 (en) 2018-08-22 2025-04-15 The Regents Of The University Of California Variant type V CRISPR/Cas effector polypeptides and methods of use thereof
WO2020247883A2 (en) 2019-06-07 2020-12-10 Scribe Therapeutics Inc. Deep mutational evolution of biomolecules
EP3980533A1 (en) 2019-06-07 2022-04-13 Scribe Therapeutics Inc. Engineered casx systems
US12454685B2 (en) 2019-07-08 2025-10-28 The Regents Of The University Of California Variant type V CRISPR/Cas effector polypeptides and methods of use thereof
CN114340656B (zh) 2019-08-02 2024-07-30 孟山都技术公司 使用huh内切核酸酶促进靶向基因组修饰的方法和组合物
WO2021050593A1 (en) 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for the targeting of sod1
WO2021050601A1 (en) 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy
WO2021084533A1 (en) 2019-10-28 2021-05-06 Targetgene Biotechnologies Ltd Pam-reduced and pam-abolished cas derivatives compositions and uses thereof in genetic modulation
EP4069716A1 (en) * 2019-12-06 2022-10-12 Scribe Therapeutics Inc. Particle delivery systems
WO2021113763A1 (en) 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
EP4069846A1 (en) * 2019-12-07 2022-10-12 Scribe Therapeutics Inc. Compositions and methods for the targeting of htt
IL294620A (en) 2020-01-10 2022-09-01 Scribe Therapeutics Inc Compositions and methods for the targeting of pcsk9
AU2021237633A1 (en) 2020-03-18 2022-10-06 Scribe Therapeutics Inc. Compositions and methods for the targeting of C9orf72
WO2022119957A1 (en) 2020-12-02 2022-06-09 The Regents Of The University Of California Crispr-cas effector polypeptides and methods of use thereof
KR20230128289A (ko) 2020-12-03 2023-09-04 스크라이브 테라퓨틱스 인크. 조작된 클래스 2 유형 v crispr 시스템
AU2021391783A1 (en) 2020-12-03 2023-06-22 Scribe Therapeutics Inc. Compositions and methods for the targeting of bcl11a
WO2022125843A1 (en) 2020-12-09 2022-06-16 Scribe Therapeutics Inc. Aav vectors for gene editing
CA3212612A1 (en) 2021-03-26 2022-09-29 Brent E. STEAD Targeting oncogentic mutations with dual-cleaving endonuclease
WO2022261149A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
GB2625500B (en) 2021-09-21 2026-04-01 Scribe Therapeutics Inc Engineered CasX repressor systems
EP4405490A2 (en) 2021-09-23 2024-07-31 Scribe Therapeutics Inc. Self-inactivating vectors for gene editing
WO2023235818A2 (en) 2022-06-02 2023-12-07 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
TW202413643A (zh) 2022-06-07 2024-04-01 美商斯奎柏治療公司 用於靶向pcsk9的組合物及方法
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
WO2023240157A2 (en) 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of dmd
WO2024206676A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of lpa

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AU2021392719A1 (en) 2023-06-22
US20250011756A1 (en) 2025-01-09
PE20231178A1 (es) 2023-08-01
US20240026385A1 (en) 2024-01-25
CL2023001594A1 (es) 2024-01-05
US12559743B2 (en) 2026-02-24
US20230054437A1 (en) 2023-02-23
MX2023006566A (es) 2023-08-07
AU2021392719A9 (en) 2024-10-03
JP2023552374A (ja) 2023-12-15
GB202309876D0 (en) 2023-08-16
WO2022120095A1 (en) 2022-06-09
US20240376462A1 (en) 2024-11-14
TW202237836A (zh) 2022-10-01
GB2616584B (en) 2026-03-04
KR20230128289A (ko) 2023-09-04
CL2024001727A1 (es) 2024-10-18
GB2616584A (en) 2023-09-13
US12163125B2 (en) 2024-12-10
AU2021392719B2 (en) 2025-08-14

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