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Gene therapy aims to fix protein deficiency in lung and liver disease

NCT ID NCT07227207

First seen Nov 12, 2025 · Last updated May 25, 2026 · Updated 22 times

Summary

This study tests an experimental gene therapy called TSRA-196 in 72 adults with a severe form of Alpha-1 Antitrypsin Deficiency (the PiZZ genotype) who have lung or liver disease. The treatment is designed to help the body produce more of the protective protein it lacks. The trial is in early phases (1 and 2) and focuses on safety, tolerability, and whether the therapy can raise protein levels to normal.

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Contacts and locations

Study contacts

  • Contact

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

Locations

  • Clinical Study Center

    RECRUITING

    Boston, Massachusetts, 02118, United States

  • Clinical Study Center

    RECRUITING

    Charleston, South Carolina, 29425, United States

  • Clinical Study Center

    RECRUITING

    Fitzroy, Victoria, Australia

Conditions

Explore the condition pages connected to this study.