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
Application number
PCT/US2023/074440
Other languages
French (fr)
Other versions
WO2024064607A9 (en
WO2024064607A2 (en
Inventor
Rafi EMMANUEL
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.
Publication of WO2024064607A2 publication Critical patent/WO2024064607A2/en
Publication of WO2024064607A3 publication Critical patent/WO2024064607A3/en
Publication of WO2024064607A9 publication Critical patent/WO2024064607A9/en

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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
    • 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 [CRISPRs]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)

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 WO2024064607A2 (en)

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 WO2024064607A2 (en) 2022-09-19 2023-09-18 Biallelic knockout of tet2

Country Status (1)

Country Link
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
WO2024064607A9 (en) 2024-07-25
WO2024064607A2 (en) 2024-03-28

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