Beam doses first patient in pivotal BEAM-302 cohort, updates PKU and sickle cell timelines

Beam Therapeutics announced on August 4, 2026 that in July, Beam dosed the first patient in the global pivotal cohort of the ongoing Phase 1/2 trial evaluating BEAM-302 in patients with AATD-associated lung disease, with or without liver disease.¹ The cohort is designed to support a potential accelerated approval path in the United States.¹ Updated clinical data for BEAM-302 were selected for a late-breaking oral presentation at the European Respiratory Society Congress, taking place September 5-9, 2026, in Barcelona, Spain.¹

In the company's hematology program, dosing is complete in all adult and adolescent patients enrolled in the Phase 1/2 BEACON trial¹ of risto-cel. Beam said it expects to report updated data for the BEACON trial by year-end 2026 and submit a biologics license application for risto-cel as early as year-end 2026.¹

For PKU, in June, Beam announced that the FDA cleared the investigational new drug application for BEAM-304.¹ Beam has initiated clinical start-up activities for the planned Phase 1/2 trial for BEAM-304, which will initially evaluate safety, tolerability, and reduction of blood Phe levels in PKU patients with the R408W mutation.¹ The company separately said it expects to report initial clinical data¹ for its GSDIa program, BEAM-301, in 2026.

On financials, Beam reported cash, cash equivalents and marketable securities of $1.2 billion as of June 30, 2026, compared to $1.2 billion as of December 31, 2025.¹ The company said this position, together with an additional $200 million expected to be drawn from its facility with Sixth Street, will fund anticipated operating expenses and capital expenditure requirements into mid-2029, funding the company through the anticipated launch of risto-cel in SCD, execution of the BEAM-302 pivotal development plan in AATD, and clinical proof of concept for BEAM-304 in PKU.¹