CP-301

CP-301 is a small molecule drug in early-stage discovery, developed by Contera Pharma in collaboration with Vernalis. It is designed as a first-in-class modulator of RNA splicing events that are critical for the development of certain diseases. The primary therapeutic focus is on Charcot-Marie-Tooth disease (CMT), a group of inherited peripheral neuropathies characterized by progressive degeneration of motor and sensory nerves. CMT leads to muscle weakness and sensory loss, primarily affecting the extremities. The mechanism aims to address genetic mutations impacting nerve structure and function by modulating RNA splicing[2][1].