Beam reports Q3 2026 results, outlines pipeline timelines for risto-cel, BEAM-302, BEAM-304

Beam Therapeutics reported third quarter 2026 financial results and clinical updates on November 3, 2026. The company said it expects to report updated clinical data by year-end 2026 and submit a U.S. biologics license application (BLA) for risto-cel as early as year-end 2026.¹

For BEAM-302 in alpha-1 antitrypsin deficiency, Beam presented data at the European Respiratory Society Congress in September showing, with up to 18 months of follow-up, that a single dose produced lasting gains in total and functional AAT, lower circulating neutrophil elastase activity, and reduced mutant Z-AAT and toxic Z-polymers, according to the company reinforcing its potential as a one-time treatment for both the lung and liver manifestations of AATD, with up to 18 months of follow-up, the data demonstrated durable increases in total and functional AAT, decreased circulating neutrophil elastase activity, and reductions in mutant Z-AAT and toxic circulating Z-polymers following a single dose.¹ Enrollment and dosing continue in the global pivotal cohort of the ongoing Phase 1/2 trial, which is designed to support a potential accelerated approval path in the United States, with dosing completion expected in the first half of 2027.¹

For BEAM-304 in phenylketonuria, Beam nominated a third base editor targeting an undisclosed PKU mutation, and the company believes the program could now address up to 50% of U.S. PKU patients across its three editors.¹ Dosing in the Phase 1/2 trial is expected to begin in early 2027.¹

For BEAM-301 in glycogen storage disease type Ia, data presented at the European Society of Gene & Cell Therapy Congress in October showed dose-dependent improvements in time to fasting hypoglycemia across all patients, with two of three patients in the 40 mg cohort reaching the protocol-defined maximum controlled fasting duration of 15 hours or more, and complete elimination of starch supplementation.¹ Beam is now enrolling a 60 mg cohort and plans to engage the FDA on a potential approval path.¹

On finances, cash, cash equivalents and marketable securities totaled $1.0 billion as of September 30, 2026, down from $1.2 billion at the end of 2025.¹ Combined with an additional $200 million expected under its Sixth Street credit facility, Beam said this funds operations into mid-2029, covering a potential risto-cel launch, the BEAM-302 pivotal plan, and BEAM-304 proof of concept.¹