Salvador Rico, MD, PhD, on A Multi-Pronged Approach to Tackling Dravet Syndrome


The chief medical officer of Encoded Therapeutics discussed ETX101 gene therapy and other initiatives in the company’s overall Dravet program.

“We've been working on this program for a few years now. And in parallel to all the body of preclinical data that we've generated, we have also been working on different initiatives, including the largest natural history study that has been conducted today that we call ENVISION... In parallel to that is an initiative that is patient focused or caregiver focused, called Dravet ENGAGE to understand the needs, the perspectives and the ones of the patient community, as well as a biomarker discovery effort called ELUCIDATE."

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

CGTLive spoke with Salvador Rico, MD, PhD, chief medical officer of Encoded Therapeutic, to learn more about the upcoming trials as well as the larger program the company is working on to better understand and treat Dravet syndrome. This program includes the 2 new 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. It also consists of the natural history ENVISION (NCT04537832) study, the biomarker discovery ELUCIDATE program, and the patient/caregiver focused Dravet ENGAGE study. Rico also spoke about Dravet syndrome and how ETX101 may potentially upregulate the healthy allele in patients with SCN1A+ Dravet syndrome and correct the pathophysiology of the disease.

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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