Encoded Therapeutics is shrinking its headcount by 29% as the gene therapy biotech draws resources away from its early-stage R&D toward its Dravet syndrome program.
The asset in question, dubbed ETX101, is an AAV9-based therapy designed to upregulate the expression of the SCN1A gene in inhibitory neurons. Encoded hopes ETX101 could become the first one-time therapy to address the underlying genetic cause of Dravet syndrome.
So far, eight infants and children between six months and seven years old have been dosed as part of a program spanning the U.S., U.K. and Australia.
With no treatment-related adverse events reported to date and a preliminary efficacy readout penciled in for the second half of this year, the program is now Encoded's top priority. However, the central nervous system-focused company is also planning to submit a request with the FDA next year to take ETX201, its AAV9-based vectorized microRNA (miRNA) therapy for Angelman syndrome, into the clinic.
Further back in preclinical development are AAV9-based vectorized miRNA candidates aimed at chronic pain and Alzheimer’s.
“Given the significant potential of our portfolio, Encoded is focusing resources on ETX101 and our established programs where we have the greatest opportunity to create near-term value,” Encoded CEO Kartik Ramamoorthi, Ph.D., said in the release. “In parallel, we have made the difficult decision to reduce the size of our technology and early-stage research and development functions.”
The resulting 29% layoffs should extend Encoded’s cash runway into the third quarter of 2026, the company explained in the release. It’s not the first time the biotech has resorted to layoffs, with Encoded letting go of 10% of its workforce in 2023 after assessing its spend.
Drug products for all of Encoded’s various ongoing programs will be manufactured in-house at a GMP facility in North Carolina's Research Triangle Park that the biotech expects to become fully operational in the first quarter of this year.