XoNovo is a preclinical-stage biopharmaceutical company engaged in the development of small-molecule therapeutics for preventing the progressive loss of neuronal function across multiple neurodegenerative diseases.
The company's short-term focus is on its lead drug, XN001, in support of filing an investigational new drug application for Batten disease (BD), also known as neuronal ceroid lipofuscinoses, a fatal neurodegenerative orphan disorder.
XoNovo utilizes a novel approach of selective modulation of autophagy and axonal transport machinery in neurons. XN001 has been extensively tested in both in-vitro and in-vivo models, showing promising results in neurodegenerative diseases including BD, Alzheimer's disease, glioblastoma, muscular dystrophy, and tuberous sclerosis.