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WO2023227669A3 - Novel nucleic acid-editing proteins - Google Patents

Novel nucleic acid-editing proteins Download PDF

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Publication number
WO2023227669A3
WO2023227669A3 PCT/EP2023/063933 EP2023063933W WO2023227669A3 WO 2023227669 A3 WO2023227669 A3 WO 2023227669A3 EP 2023063933 W EP2023063933 W EP 2023063933W WO 2023227669 A3 WO2023227669 A3 WO 2023227669A3
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acid
novel nucleic
novel
editing proteins
editing
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.)
Ceased
Application number
PCT/EP2023/063933
Other languages
French (fr)
Other versions
WO2023227669A2 (en
Inventor
Tyson David BOWEN
Lila Herk RIEBER
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.)
UCB Biopharma SRL
Original Assignee
UCB Biopharma SRL
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 UCB Biopharma SRL filed Critical UCB Biopharma SRL
Priority to JP2024569521A priority Critical patent/JP2025517515A/en
Priority to EP23729980.5A priority patent/EP4532704A2/en
Publication of WO2023227669A2 publication Critical patent/WO2023227669A2/en
Publication of WO2023227669A3 publication Critical patent/WO2023227669A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005Cytidine deaminase (3.5.4.5)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Pulmonology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)

Abstract

The present invention provides novel base-editing systems comprising an RNA-guided nuclease domain and novel cytidine deaminase domain. Novel uracil protecting peptides useful for base editing applications are also provided.
PCT/EP2023/063933 2022-05-26 2023-05-24 Novel nucleic acid-editing proteins Ceased WO2023227669A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2024569521A JP2025517515A (en) 2022-05-26 2023-05-24 Novel nucleic acid editing protein
EP23729980.5A EP4532704A2 (en) 2022-05-26 2023-05-24 Novel nucleic acid-editing proteins

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263346065P 2022-05-26 2022-05-26
US63/346,065 2022-05-26

Publications (2)

Publication Number Publication Date
WO2023227669A2 WO2023227669A2 (en) 2023-11-30
WO2023227669A3 true WO2023227669A3 (en) 2024-02-22

Family

ID=86760329

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2023/063933 Ceased WO2023227669A2 (en) 2022-05-26 2023-05-24 Novel nucleic acid-editing proteins

Country Status (3)

Country Link
EP (1) EP4532704A2 (en)
JP (1) JP2025517515A (en)
WO (1) WO2023227669A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12454694B2 (en) 2018-09-07 2025-10-28 Beam Therapeutics Inc. Compositions and methods for improving base editing

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020081568A1 (en) * 2018-10-15 2020-04-23 University Of Massachusetts Programmable dna base editing by nme2cas9-deaminase fusion proteins
WO2022008466A1 (en) * 2020-07-06 2022-01-13 Wageningen Universiteit Base editing tools
WO2022015969A1 (en) * 2020-07-15 2022-01-20 LifeEDIT Therapeutics, Inc. Uracil stabilizing proteins and active fragments and variants thereof and methods of use

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993025673A1 (en) 1992-06-04 1993-12-23 The Regents Of The University Of California In vivo gene therapy with intron-free sequence of interest
CA2153593C (en) 1993-01-26 2010-04-13 David B. Weiner Compositions and methods for delivery of genetic material
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
US5962428A (en) 1995-03-30 1999-10-05 Apollon, Inc. Compositions and methods for delivery of genetic material
US6768550B2 (en) 2002-07-26 2004-07-27 Proterion Corporation Beam shifting surface plasmon resonance system and method
WO2017070633A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Evolved cas9 proteins for gene editing

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020081568A1 (en) * 2018-10-15 2020-04-23 University Of Massachusetts Programmable dna base editing by nme2cas9-deaminase fusion proteins
WO2022008466A1 (en) * 2020-07-06 2022-01-13 Wageningen Universiteit Base editing tools
WO2022015969A1 (en) * 2020-07-15 2022-01-20 LifeEDIT Therapeutics, Inc. Uracil stabilizing proteins and active fragments and variants thereof and methods of use

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
DATABASE EMBL [online] 7 April 2016 (2016-04-07), YUAN Y: "Bacillus phage vB_BpuM-BpSp uracil-DNA glycosylase inhibitor", XP093076687, retrieved from EBI accession no. ALN97938 Database accession no. ALN97938 *
DATABASE EMBLWGS [online] 31 January 2021 (2021-01-31), HARRISON L H: "Serratia marcescens hypothetical protein", XP093076675, retrieved from EBI accession no. MBN5231334 Database accession no. MBN5231334.1 *
DATABASE Geneseq [online] 30 June 2022 (2022-06-30), "Cytidine deaminase (APG30130), SEQ ID 86.", XP093076574, retrieved from EBI accession no. GSP:BKO08826 Database accession no. BKO08826 *
DATABASE Geneseq [online] 30 March 2022 (2022-03-30), "Cytidine deaminase (APG30125), SEQ ID 81.", XP093115791, retrieved from EBI accession no. GSP:BKO08821 Database accession no. BKO08821 *
DATABASE TrEMBL [online] 31 July 2019 (2019-07-31), GOEKER M.: "Genomic Encyclopedia of Type Strains, Phase IV (KMG-IV): sequencing theRT most valuable type-strain genomes for metagenomic binning, comparativeRT biology and taxonomic classification", XP093115800, retrieved from UniProt accession no. A0A4V3BN72_9RHOO Database accession no. A0A4V3BN72 *
KOMOR ALEXIS C ET AL: "Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage", NATURE, NATURE PUBLISHING GROUP UK, LONDON, vol. 533, no. 7603, 19 May 2016 (2016-05-19), pages 420 - 424, XP037336614, ISSN: 0028-0836, DOI: 10.1038/NATURE17946 *
WANG H.-C. ET AL: "Staphylococcus aureus protein SAUGI acts as a uracil-DNA glycosylase inhibitor", NUCLEIC ACIDS RESEARCH, vol. 42, no. 2, 22 October 2013 (2013-10-22), GB, pages 1354 - 1364, XP055855618, ISSN: 0305-1048, DOI: 10.1093/nar/gkt964 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12454694B2 (en) 2018-09-07 2025-10-28 Beam Therapeutics Inc. Compositions and methods for improving base editing

Also Published As

Publication number Publication date
EP4532704A2 (en) 2025-04-09
WO2023227669A2 (en) 2023-11-30
JP2025517515A (en) 2025-06-05

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