One-Shot gene therapy aims to stop debilitating swelling attacks

NCT ID NCT05120830

First seen Apr 01, 2026 · Last updated Apr 22, 2026 · Updated 4 times

Summary

This early-stage study is testing a single-dose gene therapy called NTLA-2002 in adults with hereditary angioedema (HAE), a genetic disorder that causes sudden, severe swelling. The main goals are to see if the treatment is safe and if it can reduce the number of swelling attacks people experience. Researchers will also measure how the drug works in the body and its effect on a key protein involved in HAE attacks.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for HEREDITARY ANGIOEDEMA are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Locations

  • Clinical Trial Site

    Campbelltown, Australia

  • Clinical Trial Site

    Grenoble, France

  • Clinical Trial Site

    Lille, France

  • Clinical Trial Site

    Paris, France

  • Clinical Trial Site

    Berlin, Germany

  • Clinical Trial Site

    Frankfurt, Germany

  • Clinical Trial Site

    Amsterdam, Netherlands

  • Clinical Trial Site

    Auckland, New Zealand

  • Clinical Trial Site

    Cambridge, United Kingdom

Conditions

Explore the condition pages connected to this study.