Removing barriers to recovery after neurotrauma
BioAxone’s scientific team has unmatched knowledge and a deep understanding of Rho signaling pathways, axon regeneration, and neurotrauma. This knowledge and understanding emanates from over 20 years of research led by our founder into the key signaling pathways and proteins that inhibit axon regeneration after spinal cord injury. The spinal cord is not hard wired and “learning” can occur in spinal cord circuits. Drugs that promote axon regeneration stimulate natural repair processes, and maximizing function after neurotrauma is most achievable with drugs that enhance connectivity. BioAxone’s work has demonstrated that Rho is a key target to impact regeneration and repair.
A second generation drug in development that promotes axon regeneration is a self-delivering RNAi to supres expression of PTEN. PTEN has been identified as an important intrinsic barrier to axon regeneration.
Our small molecule drug candidates, Rho kinase (ROCK) inhibitors, inhibit a downstream target of Rho. They have the potential to repair a broken blood brain barrier. They have been out-licensed to Neurelis for continued development.
ROCK is hyper-activated in endothelial cells in neurovascular disorders, including the disease of cerebral cavernous malformation (CCM), also called cavernous angioma, a serious genetic disease where endothelial lesions in the brain become leaky and bleed. There is no treatment for CCM except invasive surgery. ROCK is a target for treatment because the genetic mutation leading to angioma causes ROCK hyper-activation in brain capillary endothelial cells. Treatments effective for CCM also have utility in treating the blood brain defects in other neurovascular disorders, including stroke.