YOLT-101

YOLT-101 is an investigational in vivo base editing therapy developed by YolTech Therapeutics for the treatment of familial hypercholesterolemia (FH), a genetic disorder characterized by high levels of low-density lipoprotein cholesterol (LDL-C) due to mutations affecting cholesterol metabolism. The drug uses advanced gene editing technology delivered via lipid nanoparticles (LNPs) to target and permanently deactivate the PCSK9 gene in liver cells, aiming to reduce LDL-C levels. Preclinical studies have shown that a single dose can significantly lower LDL-C for up to two years in non-human primate models. YOLT-101 is designed as a potentially curative, one-time treatment and is currently being evaluated in clinical trials for safety, tolerability, and efficacy[1][2][3][4][5].