GA-003 (Genosera)

GA-003 is a preclinical gene therapy candidate developed by Genosera for the treatment of Lysosomal Acid Lipase Deficiency (LAL-D). LAL-D is a rare, progressive lysosomal storage disorder caused by mutations in the *LIPA* gene, which leads to a deficiency in the lysosomal acid lipase enzyme. This enzyme is responsible for breaking down cholesteryl esters and triglycerides; its absence results in toxic lipid accumulation in the liver, spleen, and other organs. GA-003 is designed to provide a functional *LIPA* gene to restore enzyme production and prevent organ damage. Genosera expects to initiate pre-IND discussions with the FDA in 2026, with clinical entry targeted for 2027.