[go: up one dir, main page]

WO2022008557A3 - Modulation of cftr expression - Google Patents

Modulation of cftr expression Download PDF

Info

Publication number
WO2022008557A3
WO2022008557A3 PCT/EP2021/068748 EP2021068748W WO2022008557A3 WO 2022008557 A3 WO2022008557 A3 WO 2022008557A3 EP 2021068748 W EP2021068748 W EP 2021068748W WO 2022008557 A3 WO2022008557 A3 WO 2022008557A3
Authority
WO
WIPO (PCT)
Prior art keywords
expression
modulation
cftr
cftr expression
cftr gene
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/EP2021/068748
Other languages
French (fr)
Other versions
WO2022008557A2 (en
Inventor
Ami M. Kabadi
Timothy E. Reddy
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
Publication of WO2022008557A2 publication Critical patent/WO2022008557A2/en
Publication of WO2022008557A3 publication Critical patent/WO2022008557A3/en
Anticipated expiration legal-status Critical
Ceased 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
    • 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
    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The present invention provides a DNA targeting system for modulating the expression of the CFTR gene. The present invention utilizes CRISPR/Cas9-based epigenome editing tools for repression or activation of CFTR gene expression and provides the regions of the CFTR gene that can be targeted by such system to increase the CFTR expression.
PCT/EP2021/068748 2020-07-08 2021-07-07 Modulation of cftr expression Ceased WO2022008557A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063049330P 2020-07-08 2020-07-08
US63/049,330 2020-07-08

Publications (2)

Publication Number Publication Date
WO2022008557A2 WO2022008557A2 (en) 2022-01-13
WO2022008557A3 true WO2022008557A3 (en) 2022-02-17

Family

ID=76920782

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2021/068748 Ceased WO2022008557A2 (en) 2020-07-08 2021-07-07 Modulation of cftr expression

Country Status (1)

Country Link
WO (1) WO2022008557A2 (en)

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015179656A2 (en) * 2014-05-23 2015-11-26 The Scripps Research Institute Specific targeted activation of cystic fibrosis transmembrane conductance regulator (cftr)
WO2016011080A2 (en) * 2014-07-14 2016-01-21 The Regents Of The University Of California Crispr/cas transcriptional modulation
US20170022507A1 (en) * 2014-04-09 2017-01-26 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
WO2018031762A1 (en) * 2016-08-10 2018-02-15 Duke University Compositions, systems and methods for programming immune cell function through targeted gene regulation
KR20180065414A (en) * 2016-12-07 2018-06-18 연세대학교 산학협력단 Guide RNA sequences for CFTR gene deletion, T84 cell having mutated CFTR protein and use thereof
US10676726B2 (en) * 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
WO2020118073A1 (en) * 2018-12-05 2020-06-11 Vertex Pharmaceuticals Incorporated Gene-editing systems for editing a cystic fibrosis transmembrane regulator (cftr) gene

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4528493A (en) 1992-06-04 1994-01-04 Regents Of The University Of California, The In vivo gene therapy with intron-free sequence of interest
JP7085716B2 (en) 2013-06-05 2022-06-17 デューク ユニバーシティ RNA Guide Gene Editing and Gene Regulation

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170022507A1 (en) * 2014-04-09 2017-01-26 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
WO2015179656A2 (en) * 2014-05-23 2015-11-26 The Scripps Research Institute Specific targeted activation of cystic fibrosis transmembrane conductance regulator (cftr)
WO2016011080A2 (en) * 2014-07-14 2016-01-21 The Regents Of The University Of California Crispr/cas transcriptional modulation
US10676726B2 (en) * 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
WO2018031762A1 (en) * 2016-08-10 2018-02-15 Duke University Compositions, systems and methods for programming immune cell function through targeted gene regulation
KR20180065414A (en) * 2016-12-07 2018-06-18 연세대학교 산학협력단 Guide RNA sequences for CFTR gene deletion, T84 cell having mutated CFTR protein and use thereof
WO2020118073A1 (en) * 2018-12-05 2020-06-11 Vertex Pharmaceuticals Incorporated Gene-editing systems for editing a cystic fibrosis transmembrane regulator (cftr) gene

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
CHUNG WOO YOUNG ET AL: "Generation of [Delta]F508-CFTR T84 cell lines by CRISPR/Cas9-mediated genome editing", 29 August 2016, BIOTECHNOLOGY LETTERS, KLUWER ACADEMIC PUBLISHERS, DORDRECHT, PAGE(S) 2023 - 2034, ISSN: 0141-5492, XP036083395 *
ISAAC B HILTON ET AL: "Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers", NATURE BIOTECHNOLOGY, vol. 33, no. 5, 6 April 2015 (2015-04-06), New York, pages 510 - 517, XP055327077, ISSN: 1087-0156, DOI: 10.1038/nbt.3199 *
VAN KONINGSBRUGGEN-RIETSCHEL SILKE: "Novel treatment modalities for cystic fibrosis", 9 January 2019, DER PNEUMOLOGE, SPRINGER MEDIZIN, HEIDELBERG, PAGE(S) 88 - 97, ISSN: 1613-5636, XP036711343 *

Also Published As

Publication number Publication date
WO2022008557A2 (en) 2022-01-13

Similar Documents

Publication Publication Date Title
MX2022012110A (en) Class ii, type ii crispr systems.
WO2021178720A3 (en) Methods and compositions for modulating a genome
MX2020006072A (en) Cpf1-related methods and compositions for gene editing.
EP4495235A3 (en) Grna fusion molecules, gene editing systems, and methods of use thereof
WO2018049009A3 (en) Modulation of liver genes
WO2020236972A3 (en) Non-classi engineered crispr-cas polynucleotide targeting system
WO2011100374A3 (en) Mediator and cohesin connect gene expression and chromatin architecture
WO2018195439A3 (en) K-ras modulators
MX2021008487A (en) Targeted nuclear rna cleavage and polyadenylation with crispr-cas.
MY201938A (en) Methods and compositions for modulating splicing
WO2021207530A8 (en) Compounds and methods for modulating splicing
WO2017180694A8 (en) Cas9 fusion molecules, gene editing systems, and methods of use thereof
WO2019209885A3 (en) Enzyme engineering to alter the functional repertoire of cannabinoid synthases
CR20220566A (en) Compounds and methods for modulating splicing
MX2022014126A (en) Enpp1 modulators and uses thereof.
WO2021203016A3 (en) Protein-protein interaction stabilizers
WO2020186059A3 (en) Novel high fidelity rna-programmable endonuclease systems and uses thereof
WO2018169901A8 (en) Synthetic promoters
WO2019023654A3 (en) Discovery of small molecules that target the androgen receptor and uses thereof
WO2022232638A3 (en) Enzymes with ruvc domains
WO2021041001A3 (en) AUGMENTED sgRNAS AND METHODS FOR THEIR USE TO ENHANCE SOMATIC AND GERMLINE PLANT GENOME ENGINEERING
MX2022010681A (en) Compounds and methods for modulating splicing.
WO2022232016A3 (en) Chimeric receptors targeting adgre2 and/or clec12a and uses thereof
MX2024002930A (en) Methods and compositions for modulating a genome.
WO2022008557A3 (en) Modulation of cftr expression

Legal Events

Date Code Title Description
NENP Non-entry into the national phase

Ref country code: DE

122 Ep: pct application non-entry in european phase

Ref document number: 21742103

Country of ref document: EP

Kind code of ref document: A2