One-Time gene fix aims to shield lungs in rare disease

NCT ID NCT06996756

First seen Oct 31, 2025 · Last updated May 01, 2026 · Updated 24 times

Summary

This early-stage study tests a gene therapy for people with alpha-1 antitrypsin deficiency, a condition that can damage the lungs. The treatment uses a single IV dose to help the body make a protective protein. Researchers are checking safety and whether it works in 16 adults with mild to moderate lung disease.

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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.