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AU2024261906A1 - Modified guide rna - Google Patents

Modified guide rna

Info

Publication number
AU2024261906A1
AU2024261906A1 AU2024261906A AU2024261906A AU2024261906A1 AU 2024261906 A1 AU2024261906 A1 AU 2024261906A1 AU 2024261906 A AU2024261906 A AU 2024261906A AU 2024261906 A AU2024261906 A AU 2024261906A AU 2024261906 A1 AU2024261906 A1 AU 2024261906A1
Authority
AU
Australia
Prior art keywords
modified
target
guide rna
modified guide
present
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
AU2024261906A
Inventor
Brian CAFFERTY
Ho YAU
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.)
Beam Therapeutics Inc
Original Assignee
Beam 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 Beam Therapeutics Inc filed Critical Beam Therapeutics Inc
Publication of AU2024261906A1 publication Critical patent/AU2024261906A1/en
Pending legal-status Critical Current

Links

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
    • 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
    • C12N2310/34—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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The present invention relates to modified gRNA molecules, compositions and methods for site specific gene editing and genomic modification, such as DNA cleavage, and gene activation or repression. The present modified guide RNAs have modified secondary structure (e.g., long upper stem and a modified hairpin structure), which target the target DNA sequence specifically with reduced off-target activity.
AU2024261906A 2023-04-28 2024-04-26 Modified guide rna Pending AU2024261906A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202363462873P 2023-04-28 2023-04-28
US63/462,873 2023-04-28
PCT/US2024/026601 WO2024227047A2 (en) 2023-04-28 2024-04-26 Modified guide rna

Publications (1)

Publication Number Publication Date
AU2024261906A1 true AU2024261906A1 (en) 2025-10-23

Family

ID=91334660

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2024261906A Pending AU2024261906A1 (en) 2023-04-28 2024-04-26 Modified guide rna

Country Status (2)

Country Link
AU (1) AU2024261906A1 (en)
WO (1) WO2024227047A2 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7875733B2 (en) 2003-09-18 2011-01-25 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising 4′-thionucleosides for use in gene modulation
ATE232880T1 (en) 1996-11-18 2003-03-15 Takeshi Imanishi NEW NUCLEOTIDE ANALOGUES
JP3756313B2 (en) 1997-03-07 2006-03-15 武 今西 Novel bicyclonucleosides and oligonucleotide analogues
SI20474A (en) 1998-05-26 2001-08-31 Icn Pharmaceuticals, Inc. Novel nucleosides having bicyclic sugar moiety
IL145495A0 (en) 1999-03-18 2002-06-30 Exiqon As Xylo-lna analogues
AU3418800A (en) 1999-03-24 2000-10-09 Exiqon A/S Improved synthesis of (2.2.1)bicyclo nucleosides
HK1048322A1 (en) 1999-05-04 2003-03-28 埃克西库恩公司 L-ribo-lna analogues
WO2001000641A1 (en) 1999-06-25 2001-01-04 Sankyo Company, Limited Novel bicyclonucleoside derivatives
JP4151751B2 (en) 1999-07-22 2008-09-17 第一三共株式会社 New bicyclonucleoside analogues
US7696345B2 (en) 2002-11-05 2010-04-13 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
JP4731324B2 (en) 2003-08-28 2011-07-20 武 今西 N-O bond cross-linked novel artificial nucleic acid
DK2314594T3 (en) 2006-01-27 2014-10-27 Isis Pharmaceuticals Inc 6-modified bicyclic nucleic acid analogues
EP2125852B1 (en) 2007-02-15 2016-04-06 Ionis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
AU2008260277C1 (en) 2007-05-30 2014-04-17 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
ATE538127T1 (en) 2007-07-05 2012-01-15 Isis Pharmaceuticals Inc 6-DISUBSTITUTED BICYCLIC NUCLEIC ACID ANALOGUES
AU2015355546B2 (en) * 2014-12-03 2021-10-14 Agilent Technologies, Inc. Guide RNA with chemical modifications
WO2017044776A1 (en) * 2015-09-10 2017-03-16 Texas Tech University System Single-guide rna (sgrna) with improved knockout efficiency
EP3159407A1 (en) * 2015-10-23 2017-04-26 Silence Therapeutics (London) Ltd Guide rnas, methods and uses
WO2017070633A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Evolved cas9 proteins for gene editing
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
CN110291198B (en) * 2016-12-08 2024-11-26 因特利亚治疗公司 Modified guide RNA
US11939575B2 (en) * 2017-12-19 2024-03-26 City Of Hope Modified tracrRNAs gRNAs, and uses thereof
WO2019183000A1 (en) * 2018-03-19 2019-09-26 University Of Massachusetts Modified guide rnas for crispr genome editing
EP4069838A1 (en) * 2019-12-03 2022-10-12 Beam Therapeutics Inc. Synthetic guide rna, compositions, methods, and uses thereof
CN117529555A (en) 2021-03-23 2024-02-06 比姆医疗股份有限公司 Novel CRISPR enzymes, methods, systems and uses thereof
EP4095246A1 (en) * 2021-05-27 2022-11-30 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Novel crispr grnas
AU2022413670A1 (en) 2021-12-17 2024-07-04 Beam Therapeutics Inc. Crispr enzymes, methzods, systems and uses thereof
EP4540379A2 (en) * 2022-06-15 2025-04-23 Beam Therapeutics Inc. Compositions and methods for reducing complement activation
WO2024006772A2 (en) * 2022-06-27 2024-01-04 Beam Therapeutics Inc. Adenosine deaminase base editors and methods for use thereof
JP2025529915A (en) * 2022-08-25 2025-09-09 ライフ エディット セラピューティクス，インコーポレイティド Chemical modification of guide RNA with locked nucleic acids for RNA-guided nucleic acid-mediated gene editing

Also Published As

Publication number Publication date
WO2024227047A3 (en) 2024-12-19
WO2024227047A2 (en) 2024-10-31

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