Gene therapy for adults with alpha-1 antitrypsin deficiency
Part of Digestive system, Genetic & congenital, Lungs & breathing clinical trials.
This study tests a one-time gene therapy called BEAM-302 that aims to fix the faulty gene causing alpha-1 antitrypsin deficiency (AATD), a condition that damages the lungs and liver. The goal is to improve AAT levels and slow or stop disease progression.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 75 years old.
- You have a confirmed PiZZ genetic type of AATD.
- Your blood AAT level is low (below 11 μM) for parts A and C.
- You have at least moderate lung function (FEV1 above 30-40% depending on the part) and evidence of emphysema on imaging for parts A and C.
- You have not had a lung or liver transplant and are not on a waiting list for one.
- You can stop taking augmentation therapy if required by the study.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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