Our lead program is a first-in-class FALCON siRNA therapeutic called DTx-1252 for Charcot-Marie-Tooth Disease Type 1A (CMT1A)
CMTA is the most common inherited neuromuscular disease affecting 150,000 patients in the US and EU. There are no approved therapies. CMT1A is driven by overexpression of the PMP22 gene in Schwann cells, which prevents myelination of peripheral nerves and causes symptoms including progressive muscle wasting, neuropathic pain, difficulty walking and, eventually for many, an inability to live independently, leading to a significant and debilitating impact on patients’ lives.
Use the slider below to see how CMT1A affects healthy nerves
Our lead candidate, DTx-1252, reverses CMT1A
By repressing PMP22, our lead candidate DTx-1252 reverses CMT1A in a mouse model that faithfully recapitulates the genetic and clinical manifestations of the disease. DTx-1252 treatment induces remyelination of axons to normal levels and increases muscle mass, grip strength, coordination and agility.
