DTx Pharma is currently applying FALCON to treat peripheral nervous system, muscular and CNS disorders, with plans to expand to additional therapeutic areas. Each of our programs follows the principles of our R&D strategy: Selecting targets that are the genetic cause of the disease, engineering potent FALCON siRNAs to repress the disease-causing gene in the relevant target tissues, and guiding clinical development through the discovery and use of biomarkers.
Our lead program is a first-in-class FALCON siRNA therapeutic called DTx-1252 for Charcot-Marie-Tooth Disease Type 1A (CMT1A).
CMT1A 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 patient lives.
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.
We anticipate moving DTx-1252 into clinical development in 2023.Learn More