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Beam Therapeutics reports promising phase I/II data for BEAM-302 in AATD, showing strong efficacy, tolerability and potential as a one-time curative treatment.

Beam Therapeutics Inc. (BEAM) Discusses Topline Phase I/II Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency Transcript

BEAM-302 is Beam's lead genetic disease program for liver-targeting therapy for alpha-1 antitrypsin deficiency (AATD) that addresses the underlying pathophysiology of both liver and lung disease.

Treatment with 60 mg of BEAM-302 Led to Mean Steady-state Total AAT Level of 16.1 µM and All Patients Consistently and Durably Above the 11 µM Protective AAT Threshold with up to 12 Months of Follow-up

BEAM shares jump 23% in six months as upbeat risto-cel data, pipeline expansion and new financing strengthen confidence in its gene-editing strategy.

Beam Therapeutics Inc. is rated a Buy for its leadership in proprietary base-editing gene therapy, targeting significant unmet needs in hematology and liver diseases. BEAM's risto-cel shows superior efficacy in sickle cell disease, but commercial success hinges on overcoming conditioning protocol challenges and competing with Vertex/Crispr's head start. The liver-targeted BEAM-302 program demonstrates strong early clinical results and could establish a competitive moat, with pivotal data and BLA submission expected in 18–24 months.

Beam Therapeutics posts a narrower Q4 loss as revenues surge past estimates. It secures $500M financing, extending its cash runway into mid-2029.