AXV-201

AXV-201 is a novel gene therapy in preclinical development for the treatment of severe obesity caused by mutations in the melanocortin 4 receptor (MC4R) gene. MC4R is a key regulator of body weight, and loss-of-function mutations are the most common cause of monogenic early-onset obesity. AXV-201 uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a codon-optimized, self-complementary human MC4R transgene. In preclinical models, administration of AXV-201 prevented obesity and metabolic disease in mice lacking functional MC4R, normalizing weight trajectory and neurometabolic markers without observed safety concerns. The therapy aims to provide targeted intervention for individuals with genetic forms of severe obesity due to MC4R deficiency[1][2][3][4].