Gene therapy breakthrough? new trial targets AATD lung and liver damage

NCT ID NCT06389877

First seen Mar 27, 2026 · Last updated Apr 22, 2026 · Updated 6 times

Summary

This study tests a new gene therapy called BEAM-302 for adults with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can cause lung and liver disease. The treatment aims to fix the faulty gene to help the body produce normal levels of the protective protein. About 106 participants will receive the therapy to see if it is safe and effective.

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Contacts and locations

Study contacts

  • Contact

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

Locations

  • Clinical Study Center

    RECRUITING

    Birmingham, Alabama, 35233, United States

  • Clinical Study Center

    RECRUITING

    Boston, Massachusetts, 02215, United States

  • Clinical Study Center

    RECRUITING

    Charleston, South Carolina, 29425, United States

  • Clinical Study Center

    RECRUITING

    Adelaide, Australia

  • Clinical Study Center

    RECRUITING

    Fitzroy, Australia

  • Clinical Study Center

    RECRUITING

    Dublin, Ireland

  • Clinical Study Center

    RECRUITING

    Leiden, Netherlands

  • Clinical Study Center

    RECRUITING

    Auckland, New Zealand

  • Clinical Study Center

    RECRUITING

    Hamilton, New Zealand

  • Clinical Study Center

    RECRUITING

    London, United Kingdom

  • Clinical Study Center

    RECRUITING

    Southampton, United Kingdom

Conditions

Explore the condition pages connected to this study.