Encoded Therapeutics Inc. has announced that the U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ETX101 for the treatment of SCN1A+ Dravet syndrome. This designation follows a review of preliminary seizure data from patients enrolled in the ongoing Phase 1/2 clinical program. ETX101 is designed to enhance SCN1A expression in GABAergic neurons, addressing the underlying cause of this severe neurological disorder.
The RMAT designation is significant as it facilitates expedited development and review processes for regenerative therapies aimed at serious diseases. Encoded Therapeutics also reported that its North Carolina GMP manufacturing facility is now operational, poised to support pivotal trials anticipated in the first half of 2026. This strategic move enhances the company’s control over product quality and development timelines, reflecting its commitment to advancing innovative treatments.
As Encoded Therapeutics progresses with its POLARIS clinical development plan, which encompasses multiple ongoing trials assessing the safety and efficacy of ETX101 in young children, the company is well-positioned to leverage its regulatory designations and in-house manufacturing capabilities. The RMAT designation not only underscores the potential of ETX101 but also represents an important step in the broader landscape of gene therapies targeting complex neurological disorders.
