CA3222159A1 - Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof - Google Patents

Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof Download PDF

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Publication number
CA3222159A1
CA3222159A1 CA3222159A CA3222159A CA3222159A1 CA 3222159 A1 CA3222159 A1 CA 3222159A1 CA 3222159 A CA3222159 A CA 3222159A CA 3222159 A CA3222159 A CA 3222159A CA 3222159 A1 CA3222159 A1 CA 3222159A1
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Canada
Prior art keywords
seq
sequence
hao1
nucleotide
polypeptide
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Pending
Application number
CA3222159A
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English (en)
French (fr)
Inventor
Quinton Norman WESSELLS
Jeffrey Raymond HASWELL
Tia Marie DITOMMASO
Noah Michael Jakimo
Sejuti SENGUPTA
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Arbor Biotechnologies Inc
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Arbor Biotechnologies Inc
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Publication of CA3222159A1 publication Critical patent/CA3222159A1/en
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    • 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
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    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C12Y—ENZYMES
    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/03—Oxidoreductases acting on the CH-OH group of donors (1.1) with a oxygen as acceptor (1.1.3)
    • C12Y101/03015—(S)-2-Hydroxy-acid oxidase (1.1.3.15)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Urology & Nephrology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3222159A 2021-06-04 2022-06-03 Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof Pending CA3222159A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202163197073P 2021-06-04 2021-06-04
US63/197,073 2021-06-04
US202163225046P 2021-07-23 2021-07-23
US63/225,046 2021-07-23
US202163292889P 2021-12-22 2021-12-22
US63/292,889 2021-12-22
US202263300727P 2022-01-19 2022-01-19
US63/300,727 2022-01-19
PCT/US2022/032144 WO2022256642A2 (en) 2021-06-04 2022-06-03 Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof

Publications (1)

Publication Number Publication Date
CA3222159A1 true CA3222159A1 (en) 2022-12-08

Family

ID=82694238

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3222159A Pending CA3222159A1 (en) 2021-06-04 2022-06-03 Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof

Country Status (12)

Country Link
US (3) US20230032141A1 (https=)
EP (1) EP4347833A2 (https=)
JP (1) JP2024520691A (https=)
KR (1) KR20240034746A (https=)
AU (1) AU2022283922A1 (https=)
CA (1) CA3222159A1 (https=)
CO (1) CO2023018656A2 (https=)
IL (1) IL308803A (https=)
MX (1) MX2023014406A (https=)
PH (1) PH12023553297A1 (https=)
TW (1) TW202317763A (https=)
WO (1) WO2022256642A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4711451A1 (en) * 2023-05-11 2026-03-18 Yoltech Therapeutics Co., Ltd Cas protein, corresponding gene editing system thereof and use thereof
WO2025147604A1 (en) 2024-01-05 2025-07-10 Arbor Biotechnologies, Inc. Methods for treating primary hyperoxaluria via genetic editing of hydroxyacid oxidase 1

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3581654B1 (en) * 2013-12-27 2021-03-31 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna
WO2019178427A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
MX2021001070A (es) * 2018-07-31 2021-05-27 Intellia Therapeutics Inc Composiciones y métodos para editar el gen hidroxiácido oxidasa 1 (hao1) para tratar la hiperoxaluria primaria tipo 1 (ph1).
CN121380024A (zh) * 2020-03-31 2026-01-23 阿伯生物技术公司 包含Cas12i2变体多肽的组合物及其用途
WO2021257730A2 (en) * 2020-06-16 2021-12-23 Arbor Biotechnologies, Inc. Cells modified by a cas12i polypeptide
CN113308451B (zh) * 2020-12-07 2023-07-25 中国科学院动物研究所 工程化的Cas效应蛋白及其使用方法

Also Published As

Publication number Publication date
JP2024520691A (ja) 2024-05-24
US20240254494A1 (en) 2024-08-01
AU2022283922A9 (en) 2023-12-14
WO2022256642A3 (en) 2023-01-12
US20230212541A1 (en) 2023-07-06
AU2022283922A1 (en) 2023-12-07
PH12023553297A1 (en) 2024-03-18
CO2023018656A2 (es) 2024-01-25
KR20240034746A (ko) 2024-03-14
IL308803A (en) 2024-01-01
TW202317763A (zh) 2023-05-01
WO2022256642A2 (en) 2022-12-08
EP4347833A2 (en) 2024-04-10
US20230032141A1 (en) 2023-02-02
MX2023014406A (es) 2024-02-12
US11821012B2 (en) 2023-11-21

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