One-Shot gene therapy tested for rare lung disease

NCT ID NCT06996756

Summary

This is an early safety study for a new gene therapy designed to treat Alpha-1 Antitrypsin Deficiency, a genetic condition that can cause serious lung disease. Researchers are testing a single injection that aims to help the body produce a protective protein long-term. The study will enroll 16 people with mild to moderate lung damage to find a safe dose and see if the treatment increases protein levels in the blood and lungs.

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Contacts and locations

Study contacts

  • Contact

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

  • Contact

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

Locations

  • WCMC Department of Genetic Medicine

    RECRUITING

    New York, New York, 10021, United States

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

    Contact

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

Conditions

Explore the condition pages connected to this study.