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.
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