BEAM-301

BEAM-301 is a liver-targeting lipid nanoparticle (LNP) formulation of base editing reagents designed to correct the R83C mutation in the G6PC gene, which is the most common disease-causing mutation responsible for the most severe form of glycogen storage disease type 1a (GSD1a). GSD1a is a serious, potentially life-threatening autosomal recessive disorder characterized by impaired glucose homeostasis, leading to life-threatening fasting hypoglycemia and long-term complications affecting the liver and kidneys. There are currently no approved disease-modifying treatments for GSD1a. BEAM-301 uses in vivo base editing technology delivered via LNPs to achieve gene correction directly in liver cells, aiming to restore normal blood glucose control and improve metabolic parameters without continuous supplementation. The drug is being developed by Beam Therapeutics and is currently being evaluated in Phase 1/2 clinical trials[1][2][3][4][5][6][8].

Other names

BEAM 301BEAM301BEAM-301