05:10 PM EDT, 03/27/2025 (MT Newswires) -- Beam Therapeutics ( BEAM ) said Thursday the US Food and Drug Administration cleared its investigational new drug application for BEAM-302 to treat alpha-1 antitrypsin deficiency, an genetic disorder that affects lungs or liver.

Earlier this month, Beam disclosed initial safety and efficacy data from a global phase 1/2 study, showcasing the "first ever" clinical genetic correction of a disease-causing mutation, Giuseppe Ciaramella, president of Beam, said late Thursday.

The company plans to continue the dose-escalation portion of part A of the trial, including enrolling and dosing a fourth cohort. It expects to report further data during H2. In addition, it plans to dose the first patient in Part B, including AATD patients with mild-to-moderate liver disease in H2.

In addition to the US, the investigational formulation has received clinical trial authorization in the UK, New Zealand, Australia, the Netherlands, and Ireland.