CRISPR One-Shot injection aims to permanently fix cholesterol genes

NCT ID NCT07491172

First seen Mar 24, 2026 · Last updated Apr 13, 2026 · Updated 1 time

Summary

This is a first-in-human safety study testing a new CRISPR-based gene therapy called CTX310. It aims to permanently edit a specific gene (ANGPTL3) to lower cholesterol and other blood fats in adults whose high cholesterol doesn't respond well to existing medications. Participants will receive a single intravenous infusion, and researchers will monitor their safety and how their cholesterol levels change over time.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Research Site 1

    RECRUITING

    Adelaide, 5000, Australia

  • Research Site 10

    NOT_YET_RECRUITING

    Jacksonville, Florida, 32216, United States

  • Research Site 11

    NOT_YET_RECRUITING

    Port Orange, Florida, 32127, United States

  • Research Site 12

    NOT_YET_RECRUITING

    Portland, Oregon, 97239, United States

  • Research Site 13

    NOT_YET_RECRUITING

    High Point, North Carolina, 27260, United States

  • Research Site 14

    NOT_YET_RECRUITING

    Durham, North Carolina, 27710, United States

  • Research Site 15

    NOT_YET_RECRUITING

    Brisbane, 4064, Australia

  • Research Site 16

    NOT_YET_RECRUITING

    Brisbane, Australia

  • Research Site 17

    NOT_YET_RECRUITING

    Orlando, Florida, 32789, United States

  • Research Site 18

    NOT_YET_RECRUITING

    Cambridge, United Kingdom

  • Research Site 2

    RECRUITING

    Melbourne, 3168, Australia

  • Research Site 3

    RECRUITING

    Heidelberg, 3084, Australia

  • Research Site 4

    RECRUITING

    Christchurch, 8011, New Zealand

  • Research Site 5

    RECRUITING

    Auckland, 2025, New Zealand

  • Research Site 6

    RECRUITING

    London, SE1 1YR, United Kingdom

  • Research Site 7

    NOT_YET_RECRUITING

    Camperdown, 2050, Australia

  • Research Site 8

    NOT_YET_RECRUITING

    London, E1 1BB, United Kingdom

  • Research Site 9

    NOT_YET_RECRUITING

    Cleveland, Ohio, 44195, United States

Conditions

Explore the condition pages connected to this study.