Beam Therapeutics reports one-year data from early-stage AATD gene therapy trial

Beam Therapeutics released new results from its early-stage gene-editing program targeting alpha-1 antitrypsin deficiency (AATD), a rare condition that can damage the lungs and liver.

The data come from a Phase 1/2 study that enrolled six patients and used a base-editing approach to correct the underlying genetic defect. One year after treatment, the investigators reported that the molecular correction remained stable.

Safety signals were limited, and no new adverse events were observed during the follow-up period, suggesting the therapy continues to be well tolerated.

Beam said the findings support continued development of the candidate and will inform the design of larger trials aimed at confirming efficacy and long-term safety.