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WO2018020323A3 - Materials and methods for treatment of fatty acid disorders - Google Patents

Materials and methods for treatment of fatty acid disorders Download PDF

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Publication number
WO2018020323A3
WO2018020323A3 PCT/IB2017/001089 IB2017001089W WO2018020323A3 WO 2018020323 A3 WO2018020323 A3 WO 2018020323A3 IB 2017001089 W IB2017001089 W IB 2017001089W WO 2018020323 A3 WO2018020323 A3 WO 2018020323A3
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WO
WIPO (PCT)
Prior art keywords
methods
gene
fatty acid
coenzyme
materials
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/IB2017/001089
Other languages
French (fr)
Other versions
WO2018020323A2 (en
Inventor
Jeffrey William STEBBINS
Roman Lvovitch BOGORAD
Francine Marie GREGOIRE
Chad Albert COWAN
Ante Sven LUNDBERG
Hari Padmanabhan
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CRISPR Therapeutics AG
Original Assignee
CRISPR Therapeutics AG
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 CRISPR Therapeutics AG filed Critical CRISPR Therapeutics AG
Publication of WO2018020323A2 publication Critical patent/WO2018020323A2/en
Publication of WO2018020323A3 publication Critical patent/WO2018020323A3/en
Anticipated expiration legal-status Critical
Ceased 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
    • C12N15/10—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
    • 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
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Cell Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

The present application provides materials and methods for treating a patient with a fatty acid disorder such as medium chain acyl-coenzyme A dehydrogenase deficiency (MCADD), long-chain 3-hydroxyl-coenzyme A dehydrogenase deficiency (LCHADD), and/or very long-chain acyl-coenzyme A dehydrogenase deficiency (VLCADD) both ex vivo and in vivo. In addition, the present application provides materials and methods for genome editing to modulate the expression, function, or activity of a gene in a cell selected from the ACADM gene, the HADHA gene, and the ACADVL gene.
PCT/IB2017/001089 2016-07-25 2017-07-25 Materials and methods for treatment of fatty acid disorders Ceased WO2018020323A2 (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201662366459P 2016-07-25 2016-07-25
US201662366450P 2016-07-25 2016-07-25
US62/366,459 2016-07-25
US62/366,450 2016-07-25
US201662379036P 2016-08-24 2016-08-24
US62/379,036 2016-08-24

Publications (2)

Publication Number Publication Date
WO2018020323A2 WO2018020323A2 (en) 2018-02-01
WO2018020323A3 true WO2018020323A3 (en) 2018-03-29

Family

ID=60143731

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2017/001089 Ceased WO2018020323A2 (en) 2016-07-25 2017-07-25 Materials and methods for treatment of fatty acid disorders

Country Status (1)

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WO (1) WO2018020323A2 (en)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3054031A1 (en) * 2017-02-22 2018-08-30 Crispr Therapeutics Ag Compositions and methods for gene editing
JP7642531B2 (en) 2018-05-11 2025-03-10 ビーム セラピューティクス インク． Methods for replacing pathogenic amino acids using a programmable base editor system
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
CA3112011A1 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for delivering a nucleobase editing system
JP7631209B2 (en) * 2019-02-13 2025-02-18 ビーム セラピューティクス インク． Compositions and methods for treating glycogen storage disease type 1A
CA3163100A1 (en) * 2020-02-28 2021-09-02 Repair Biotechnologies, Inc. Targeted expression of microbial cholesterol catalysis genes reduces excess lipid
CN113430199A (en) * 2021-06-30 2021-09-24 武汉三鹰生物技术有限公司 Interfering sequence of HADHA mRNA and verification method thereof
EP4486890A1 (en) 2022-03-01 2025-01-08 CRISPR Therapeutics AG Methods and compositions for treating angiopoietin-like 3 (angptl3) related conditions

Citations (5)

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Publication number Priority date Publication date Assignee Title
WO2015048577A2 (en) * 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
WO2015070083A1 (en) * 2013-11-07 2015-05-14 Editas Medicine,Inc. CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS
US20150291966A1 (en) * 2012-07-25 2015-10-15 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
WO2016105535A2 (en) * 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Compositions and methods for manipulation of adipocyte energy consumption regulatory pathway
WO2016161380A1 (en) * 2015-04-01 2016-10-06 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy

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