Gene Edit Biotech Beam Therapeutics Touts Encouraging Data From Early-Stage Gene Therapy Study In Genetic Disorder, Raises $500 Million Via Equity

Core Insights - Beam Therapeutics Inc. announced initial safety and efficacy data from its Phase 1/2 trial of BEAM-302, showing clinical proof-of-concept for treating alpha-1 antitrypsin deficiency (AATD) and in vivo base editing [1][3] Group 1: Safety and Efficacy Data - Preliminary results from the first three single-ascending dose cohorts indicated that BEAM-302 was well tolerated, leading to durable dose-dependent correction of the disease-causing mutation [2] - Following a single infusion of BEAM-302, there were rapid, durable, and dose-dependent increases in total AAT, new production of corrected M-AAT, and decreases in mutant Z-AAT observed in circulation [3][6] Group 2: Future Plans - The company plans to continue the dose-escalation portion of Part A of the ongoing Phase 1/2 trial, including enrolling and dosing a fourth dose cohort, with further data expected to be reported at a medical conference in the second half of 2025 [4] - Beam Therapeutics intends to dose the first patient in Part B, targeting AATD patients with mild to moderate liver disease, in the second half of 2025 [4] Group 3: Financial Actions - Beam Therapeutics priced an underwritten offering of 16.15 million shares at $28.48 per share and pre-funded warrants to purchase 1.4 million at$28.47 per pre-funded warrant, with approximately $500 million in gross proceeds [5]