Salvador Rico, MD, PhD, on Developing Gene Regulation Therapy for Dravet Syndrome
The chief medical officer of Encoded Therapeutics overviews the company’s timeline and strategy for its gene regulation program for Dravet syndrome.
"What our team did is they develop an engineered transcription factor that upregulates the expression of the healthy allele in patients who have Dravet syndrome. And one important characteristic is that it's... not only gene selective, but cell selective. And we're targeting expression of these transgene in GABAergic inhibitory interneurons, because there's a whole body of literature indicating the importance of the cells in the pathophysiology of the disease. So, this is a precision medicine approach that is targeted to reestablish that balance between inhibition and excitation.”
Encoded Therapeutics is continuing to develop and evaluate its lead gene regulation therapy, ETX-101, for the potential treatment of people with SCN1A -positive Dravet syndrome. The company announced in February that it had secured clearance for clinical trials evaluating ETX-101 in this population in the United States, United Kingdom, and Australia.1,2
CGTLive® spoke with Salvador Rico, MD, PhD, chief medical officer, Encoded Therapeutics, to learn more about ETX-101 and what differentiates it from other novel genetic therapies in the investigational field. He shared that the company should soon be initiatingthe clinical trials. He also gave an overview of the company’s strategy with its Dravet program, including the clinical trials, sharing data from natural history studies that Encoded has conducted, and connecting with patient advocacy groups to better understand what endpoints represent clinically meaningful changes for patients with Dravet and better inform clinical trial design.
REFERENCES
1. Encoded Therapeutics Announces UK CTA Approval for Dravet Syndrome Gene Therapy Candidate ETX101. News release. Encoded Therapeutics. February 26, 2024. https://encoded.com/press-releases/encoded-therapeutics-announces-uk-cta-approval-for-dravet-syndrome-gene-therapy-candidate-etx101/
2. Encoded Therapeutics Announces US IND Clearance and Australian CTA Approval for Dravet Syndrome Gene Therapy Candidate ETX101. News release. Encoded Therapeutics. February 6, 2024. https://encoded.com/press-releases/encoded-therapeutics-announces-us-ind-clearance-and-australian-cta-approval-for-dravet-syndrome-gene-therapy-candidate-etx101/
OPMD and Advancements in Genetic Research
April 22nd 2025Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio, discussed the genetic basis of oculopharyngeal muscular dystrophy, as well as the primary challenges in diagnosing and treating the condition.
