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WO2024064607A3 - Biallelic knockout of tet2 - Google Patents

Biallelic knockout of tet2 Download PDF

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Publication number
WO2024064607A3
WO2024064607A3 PCT/US2023/074440 US2023074440W WO2024064607A3 WO 2024064607 A3 WO2024064607 A3 WO 2024064607A3 US 2023074440 W US2023074440 W US 2023074440W WO 2024064607 A3 WO2024064607 A3 WO 2024064607A3
Authority
WO
WIPO (PCT)
Prior art keywords
tet2
biallelic knockout
biallelic
knockout
seq
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/US2023/074440
Other languages
French (fr)
Other versions
WO2024064607A9 (en
WO2024064607A2 (en
Inventor
Rafi EMMANUEL
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.)
Emendobio Inc
Original Assignee
Emendobio 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 Emendobio Inc filed Critical Emendobio Inc
Priority to AU2023347815A priority Critical patent/AU2023347815A1/en
Priority to IL319692A priority patent/IL319692A/en
Priority to CA3267398A priority patent/CA3267398A1/en
Priority to CN202380079151.8A priority patent/CN120641567A/en
Priority to JP2025516187A priority patent/JP2025529540A/en
Priority to KR1020257010571A priority patent/KR20250072710A/en
Priority to EP23869059.8A priority patent/EP4590832A2/en
Publication of WO2024064607A2 publication Critical patent/WO2024064607A2/en
Publication of WO2024064607A3 publication Critical patent/WO2024064607A3/en
Publication of WO2024064607A9 publication Critical patent/WO2024064607A9/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • 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
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • 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
    • 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]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Compositions comprising an RNA molecule comprising a guide sequence portion having 17- 50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-37421 and methods and uses thereof.
PCT/US2023/074440 2022-09-19 2023-09-18 Biallelic knockout of tet2 Ceased WO2024064607A2 (en)

Priority Applications (7)

Application Number Priority Date Filing Date Title
AU2023347815A AU2023347815A1 (en) 2022-09-19 2023-09-18 Biallelic knockout of tet2
IL319692A IL319692A (en) 2022-09-19 2023-09-18 Biallelic knockout of tet2
CA3267398A CA3267398A1 (en) 2022-09-19 2023-09-18 Biallelic knockout of tet2
CN202380079151.8A CN120641567A (en) 2022-09-19 2023-09-18 TET2 biallelic knockout
JP2025516187A JP2025529540A (en) 2022-09-19 2023-09-18 TET2 biallelic knockout
KR1020257010571A KR20250072710A (en) 2022-09-19 2023-09-18 Biallelic knockout of TET2
EP23869059.8A EP4590832A2 (en) 2022-09-19 2023-09-18 Biallelic knockout of tet2

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263376279P 2022-09-19 2022-09-19
US63/376,279 2022-09-19

Publications (3)

Publication Number Publication Date
WO2024064607A2 WO2024064607A2 (en) 2024-03-28
WO2024064607A3 true WO2024064607A3 (en) 2024-05-30
WO2024064607A9 WO2024064607A9 (en) 2024-07-25

Family

ID=90455270

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/074440 Ceased WO2024064607A2 (en) 2022-09-19 2023-09-18 Biallelic knockout of tet2

Country Status (8)

Country Link
EP (1) EP4590832A2 (en)
JP (1) JP2025529540A (en)
KR (1) KR20250072710A (en)
CN (1) CN120641567A (en)
AU (1) AU2023347815A1 (en)
CA (1) CA3267398A1 (en)
IL (1) IL319692A (en)
WO (1) WO2024064607A2 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110236894A1 (en) * 2008-09-26 2011-09-29 Immune Disease Institute, Inc. Selective oxidation of 5-methylcytosine by tet-family proteins
US20210085720A1 (en) * 2019-05-01 2021-03-25 Pact Pharma, Inc. Compositions and methods for the treatment of cancer using a tet2 engineered t cell therapy
WO2022039565A1 (en) * 2020-08-20 2022-02-24 (주)지놈오피니언 Biomarker for diagnosing age-related macular degeneration, and use thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110236894A1 (en) * 2008-09-26 2011-09-29 Immune Disease Institute, Inc. Selective oxidation of 5-methylcytosine by tet-family proteins
US20210085720A1 (en) * 2019-05-01 2021-03-25 Pact Pharma, Inc. Compositions and methods for the treatment of cancer using a tet2 engineered t cell therapy
WO2022039565A1 (en) * 2020-08-20 2022-02-24 (주)지놈오피니언 Biomarker for diagnosing age-related macular degeneration, and use thereof

Also Published As

Publication number Publication date
EP4590832A2 (en) 2025-07-30
KR20250072710A (en) 2025-05-26
WO2024064607A9 (en) 2024-07-25
JP2025529540A (en) 2025-09-04
WO2024064607A2 (en) 2024-03-28
AU2023347815A1 (en) 2025-04-10
IL319692A (en) 2025-05-01
CA3267398A1 (en) 2024-03-28
CN120641567A (en) 2025-09-12

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