Beam begins enrollment at higher dose in gene-editing trial

Beam Therapeutics has begun enrolling a 60 mg cohort in its Phase I/II trial of BEAM-301 after initial results at 40 mg showed different responses among three patients, GEN reported. The treatment uses lipid nanoparticles to deliver gene-editing components to the liver to correct the R83C mutation associated with glycogen storage disease type Ia. At the August 17 data cutoff, two patients reached the protocol's maximum 15-hour controlled fasting duration and stopped needing starch supplementation, Beam said. The third showed no increase in time to fasting hypoglycemia, potentially because of high body mass index and heterozygosity for the mutation. Beam plans to discuss a potential approval path with the FDA.