Early Phase I/II data suggest a single infusion of BEAM-301 may restore endogenous glucose production and reduce starch dependence in patients with glycogen storage disease type Ia.
Beam Therapeutics has reported early clinical data suggesting its experimental gene-editing treatment for glycogen storage disease type Ia, or GSDIa, may help restore the body’s own glucose production after a single infusion. The findings, due to be presented at the 33rd Annual Congress of the European Society of Gene and Cell Therapy, come from an ongoing Phase I/II study of BEAM-301, a liver-targeted lipid nanoparticle therapy designed to correct the R83C mutation, one of the most common and severe disease-causing changes linked to the condition.
According to the company, six patients had received the treatment by the June 2026 data cut-off, split evenly between 20mg and 40mg dose groups. Beam said the therapy showed an acceptable safety profile that was in line with other lipid nanoparticle-based medicines. The company also reported dose-related improvements in time to fasting hypoglycaemia, the length of time patients can go without food before blood sugar falls to dangerous levels.
At baseline, the median fasting tolerance was 2.9 hours in the lower-dose group and 2.5 hours in the higher-dose group. By day 36, that had risen to 4.3 hours in the 20mg cohort, while both evaluable patients in the 40mg cohort reached the study’s maximum controlled fasting period of at least 15 hours. Beam said that pattern was consistent with restored endogenous glucose production, meaning the body appeared to be making more of its own glucose rather than relying so heavily on external starch intake.
The company also said prescribed daily starch intake fell after treatment, with reductions that ranged widely across follow-up time points, and that triglyceride levels remained broadly similar to baseline at the latest assessment. In a statement, Beam’s chief medical officer, Amy Simon, said people with GSDIa face a “relentless daily burden”, describing the need for frequent starch dosing, including overnight, to avoid life-threatening hypoglycaemia. Trial listings show BEAM-301 is being studied as a single intravenous infusion in adults with GSDIa carrying the relevant R83C variant, in a dose-escalation study that is still recruiting.
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