Encoded Therapeutics a biotechnology company focused on genetic medicines for neurological disorders, has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for its investigational therapy, ETX101. This designation was based on preliminary clinical evidence suggesting that ETX101 has the potential to address the significant unmet medical needs of patients with SCN1A+ Dravet syndrome.

ETX101 is an AAV9-based gene regulation therapy designed to increase the expression of the SCN1A gene in GABAergic inhibitory neurons, which is the underlying cause of the disorder. This RMAT designation will facilitate a closer, more frequent interaction with the FDA, and other opportunities to expedite the review process for ETX101.

The company also announced significant progress in its clinical and manufacturing efforts. Enrollment in its ongoing POLARIS dose-escalation clinical trials is expected to be completed in the fourth quarter of 2025, with plans to present interim clinical efficacy data around the same time. The POLARIS program includes a suite of trials for infants and young children across the U.S., UK, and Australia. In addition, Encoded’s new in-house GMP manufacturing facility in North Carolina is now fully operational and has begun producing ETX101. This move to internal manufacturing is a key strategic step that enhances the company's control over product quality and is expected to accelerate its development timelines. Encoded Therapeutics is also developing therapies for other neurological conditions, including Angelman syndrome, chronic pain, and Alzheimer's disease.