Beam Gets Regenerative Medicine Status for Gene Therapy

FDA has granted Beam Therapeutics a Regenerative Medicine Advanced Therapy designation for BEAM-302, a liver-targeting gene therapy designed to correct a disease-causing mutation in patients with alpha-1 antitrypsin deficiency (AATD). The inherited genetic disorder affects the lungs and/or liver, leading to early-onset emphysema and liver disease, according to the company.

The designation is based on initial safety and efficacy data from the Phase 1/2 trial of BEAM-302 that were previously reported in March, establishing clinical proof of concept as a potential treatment for AATD and in vivo base editing, the company says. “Preliminary results from the first three single-ascending dose cohorts in Part A of the study demonstrated that BEAM-302 was well tolerated, with single doses of BEAM-302 leading to durable, dose-dependent correction of the disease-causing mutation and total AAT protein levels above the therapeutic threshold in the 60 mg dose cohort,” it adds.