Osteoblast-specific HIVEP3 inhibition to treat osteoporosis

INDICATION: Osteoporosis

A team led by Guangping Gao, scientific co-founder of gene therapy plays Voyager Therapeutics Inc. and Aspa Therapeutics Inc., showed that an RNAi-based therapy inhibiting HIVEP3 in osteoblasts could treat osteoporosis. The therapy is an adeno-associated viral (AAV) serotype 9 vector encoding an miRNA against HIVEP3, with the vector's capsid protein fused to a bone-targeting peptide motif to enable osteoblast-specific inhibition. In mice with osteoporosis induced by estrogen deficiency, the gene therapy reduced markers of bone loss in the femur and lumbar vertebrae and improved femur strength and stiffness. Next steps include IND-enabling studies of bone-building AAV9 therapies with bone tropism in large animals...