Testing a new gene therapy for severe alpha-1 antitrypsin deficiency

Plain-English translation of NCT07227207 on ClinicalTrials.gov ↗ · Source last updated July 2026 · Translation generated July 2026 · How we translate trials

Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.

What is this trial?

This clinical trial is testing a new treatment called designed for people with a genetic condition called alpha-1 antitrypsin deficiency (often abbreviated as AATD). The condition occurs when the body doesn't make enough of a protective protein in the lungs and liver, which can lead to serious disease in both organs. This study will evaluate whether the medication is safe and whether it helps people with this condition.

Alpha-1 antitrypsin deficiency is a rare genetic disorder that can cause progressive, life-threatening lung and liver damage. Current treatment options are limited, so researchers are testing this new approach to help patients produce more of the missing protective protein and slow or prevent disease progression.

Do you qualify?

You likely qualify if…

you are between 18 and 70 years old
you have been diagnosed with alpha-1 antitrypsin deficiency with the PiZZ genetic type
you have lung disease (emphysema, bronchiectasis, or reduced lung function) and/or liver disease from this condition
you have not smoked for at least 6 months and agree not to smoke during the study
you have not recently taken other investigational treatments that affect alpha-1 antitrypsin levels

You likely don't qualify if…

you have HIV or hepatitis B or C infections
you have had a severe lung infection requiring hospitalization or IV antibiotics in the past year
you have had lung surgery or plan to have lung surgery during the study
you have cirrhosis or other liver disease unrelated to alpha-1 antitrypsin deficiency
you have previously received gene therapy using viral vectors

What participation looks like

You would receive the medication through an injection or infusion at a study center, with your dose potentially increased over time depending on safety results. You would have regular visits for blood tests, lung and liver function tests, and imaging scans to monitor how well the treatment works and check for side effects. The study involves three parts: an initial dose escalation phase, an expansion phase with more participants, and a follow-up phase to see how the treatment performs over time.

AI-generated summary from trial data · Jul 16, 2026 · Not medical advice

Trial locations(4 sites)

United States

Clinical Study Center, Boston, Massachusetts

Clinical Study Center, Charleston, South Carolina

Clinical Study Center, Seattle, Washington

Australia

Clinical Study Center, Fitzroy, Victoria