Encoded Therapeutics SCN9A gene therapy

Encoded Therapeutics is developing an AAV9-based microRNA (miRNA) gene therapy candidate targeting the *SCN9A* gene, which encodes the Nav1.7 voltage-gated sodium channel. Nav1.7 is a well-validated mediator of pain signaling in the peripheral nervous system. The therapy is designed to selectively and durably knock down *SCN9A* expression in dorsal root ganglion (DRG) sensory neurons via intrathecal delivery. This approach aims to provide a one-time, non-opioid treatment for chronic neuropathic pain while minimizing systemic biodistribution and potential off-target effects in the brain, heart, and liver. Preclinical data has demonstrated significant knockdown of *SCN9A* in non-human primates and durable analgesic effects in rat models of chronic pain.

Other names

SCN9A gene therapySCN-9A gene therapySCN 9A gene therapyNav1.7 gene therapyNav-1.7 gene therapyNav 1.7 gene therapyEncoded Therapeutics Chronic Pain Program