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WO2024186896A3 - Compositions and methods for epigenetic regulation of pcsk9 expression - Google Patents

Compositions and methods for epigenetic regulation of pcsk9 expression Download PDF

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Publication number
WO2024186896A3
WO2024186896A3 PCT/US2024/018670 US2024018670W WO2024186896A3 WO 2024186896 A3 WO2024186896 A3 WO 2024186896A3 US 2024018670 W US2024018670 W US 2024018670W WO 2024186896 A3 WO2024186896 A3 WO 2024186896A3
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WO
WIPO (PCT)
Prior art keywords
compositions
methods
epigenetic
epigenetic regulation
pcsk9 expression
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
PCT/US2024/018670
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French (fr)
Other versions
WO2024186896A2 (en
Inventor
Noorussahar ABUBUCKER
Ari Friedland
Morgan Maeder
Vic MYER
Frederic Tremblay
Mary Shirley MORRISON
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.)
Chroma Medicine Inc
Original Assignee
Chroma Medicine 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 Chroma Medicine Inc filed Critical Chroma Medicine Inc
Priority to AU2024230979A priority Critical patent/AU2024230979A1/en
Publication of WO2024186896A2 publication Critical patent/WO2024186896A2/en
Publication of WO2024186896A3 publication Critical patent/WO2024186896A3/en
Priority to IL323171A priority patent/IL323171A/en
Anticipated expiration legal-status Critical
Pending legal-status Critical Current

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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
    • C12N15/113—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
    • C12N15/1137—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 against enzymes
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1007—Methyltransferases (general) (2.1.1.)
    • 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
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21061—Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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

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

Abstract

This application relates to compositions and methods comprising epigenetic editors for epigenetic modification of PCSK9, as well as nucleic acids and vectors encoding the same. Also disclosed are cells epigenetic ally modified by the epigenetic editors.
PCT/US2024/018670 2023-03-06 2024-03-06 Compositions and methods for epigenetic regulation of pcsk9 expression Pending WO2024186896A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2024230979A AU2024230979A1 (en) 2023-03-06 2024-03-06 Compositions and methods for epigenetic regulation of pcsk9 expression
IL323171A IL323171A (en) 2023-03-06 2025-09-04 Compositions and methods for epigenetic regulation of pcsk9 expression

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363488736P 2023-03-06 2023-03-06
US63/488,736 2023-03-06

Publications (2)

Publication Number Publication Date
WO2024186896A2 WO2024186896A2 (en) 2024-09-12
WO2024186896A3 true WO2024186896A3 (en) 2025-02-13

Family

ID=92675704

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/018670 Pending WO2024186896A2 (en) 2023-03-06 2024-03-06 Compositions and methods for epigenetic regulation of pcsk9 expression

Country Status (3)

Country Link
AU (1) AU2024230979A1 (en)
IL (1) IL323171A (en)
WO (1) WO2024186896A2 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012158985A2 (en) * 2011-05-17 2012-11-22 Transposagen Biopharmaceuticals, Inc. Methods for site-specific genetic modification in spermatogonial stem cells using zinc finger nuclease (zfn) for the creation of model organisms
WO2014144942A2 (en) * 2013-03-15 2014-09-18 Pronai Therapeutics, Inc. Dnai for the modulation of genes
US20170137801A1 (en) * 2015-11-12 2017-05-18 Pfizer Inc. Tissue-specific genome engineering using crispr-cas9
US10465187B2 (en) * 2017-02-06 2019-11-05 Trustees Of Boston University Integrated system for programmable DNA methylation
WO2022140577A2 (en) * 2020-12-22 2022-06-30 Chroma Medicine, Inc. Compositions and methods for epigenetic editing
WO2022170007A1 (en) * 2021-02-05 2022-08-11 Dharmacon, Inc. Fusion proteins for crispr-based transcriptional repression
WO2023017004A1 (en) * 2021-08-09 2023-02-16 Cargene Therapeutics Pte. Ltd. Inhibitory nucleic acids for pcsk9

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012158985A2 (en) * 2011-05-17 2012-11-22 Transposagen Biopharmaceuticals, Inc. Methods for site-specific genetic modification in spermatogonial stem cells using zinc finger nuclease (zfn) for the creation of model organisms
WO2014144942A2 (en) * 2013-03-15 2014-09-18 Pronai Therapeutics, Inc. Dnai for the modulation of genes
US20170137801A1 (en) * 2015-11-12 2017-05-18 Pfizer Inc. Tissue-specific genome engineering using crispr-cas9
US10465187B2 (en) * 2017-02-06 2019-11-05 Trustees Of Boston University Integrated system for programmable DNA methylation
WO2022140577A2 (en) * 2020-12-22 2022-06-30 Chroma Medicine, Inc. Compositions and methods for epigenetic editing
WO2022170007A1 (en) * 2021-02-05 2022-08-11 Dharmacon, Inc. Fusion proteins for crispr-based transcriptional repression
WO2023017004A1 (en) * 2021-08-09 2023-02-16 Cargene Therapeutics Pte. Ltd. Inhibitory nucleic acids for pcsk9

Also Published As

Publication number Publication date
WO2024186896A2 (en) 2024-09-12
AU2024230979A1 (en) 2025-10-16
IL323171A (en) 2025-11-01

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