Salvador Rico, MD, PhD, on Informing Clinical Trials With Natural History Studies in Dravet


The chief medical officer of Encoded Therapeutics discussed the company’s strategy with gene therapy development for Dravet syndrome.

“One important aspect for us is to try and enable opportunities for at least some of those patients [in natural history studies] to participate in our interventional studies, not only because of our commitment with the community, but also because there's a solid body of data that would allow us to understand within the patients their change over time, once those patients receive ETX-101, and hopefully realize some benefit from their participation.”

Encoded Therapeutics has received clearances for 2 new clinical trials evaluating ETX101, its an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat SCN1A+ Dravet syndrome. The trials will be conducted in the United States (US) and Australia.

CGTLive® spoke with Salvador Rico, MD, PhD, chief medical officer of Encoded Therapeutic, to learn more about the company’s strategy for developing a gene therapy for treating Dravet syndrome, including the natural history ENVISION (NCT04537832) study, the biomarker discovery ELUCIDATE program, and the patient/caregiver focused Dravet ENGAGE study; as well as the 2 new, upcoming trials, ENDEAVOR (NCT05419492), enrolling patients aged 6 months to 3 years in the US and WAYFINDER (NCT06112275), enrolling patients aged 3 to 7 years in Australia. Rico shared how the different studies in the program work together to help inform clinical development and clinical trial assessment of gene therapy for Dravet. He also outlined ENDEAVOR and WAYFINDER and the differences between the two and their different goals.

Encoded Therapeutics Announces US IND Clearance and Australian CTA Approval for Dravet Syndrome Gene Therapy Candidate ETX101. News release. Encoded Therapeutics Inc. February 6, 2024. Accessed February 6, 2024.
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