RT001 (Revio Therapeutics)

RT001 (also known as PEPR124) is a repurposed small molecule drug being developed by Revio Therapeutics, in partnership with Peptris Technologies, for the treatment of Duchenne Muscular Dystrophy (DMD). Discovered using the AI-driven pepAI platform, RT001 is designed to upregulate utrophin, a functional paralog of dystrophin. By increasing utrophin expression, the drug aims to compensate for the loss of dystrophin in DMD patients in a mutation-agnostic manner. RT001 is formulated as an oral solution or suspension and has demonstrated efficacy in animal models by improving muscle strength and functional measures without the adverse weight-gain effects typically associated with corticosteroids. The program has received FDA Orphan Drug Designation and is currently in the preclinical stage, with plans to advance through the 505(b)(2) regulatory pathway.