Encoded Therapeutics Raises $275 Million for Genetic Medicine Pipeline

Encoded Therapeutics has raised $275 million in a new financing round to advance its genetic medicine programs for severe neurological disorders, including its lead candidate ETX101 for Dravet syndrome.

The clinical-stage biotechnology company said the financing was co-led by GV and another healthcare-focused fund. The round also included ARCH Venture Partners, Braidwell, Farallon Capital Management, Illumina Ventures, Invus, Janus Henderson Investors, Matrix Capital Management, Nolan Capital, RTW Investments, SoftBank Vision Fund 2 and Venrock.

A major portion of the funding will support the pivotal study of ETX101 in infants and young children with SCN1A-positive Dravet syndrome. Encoded also plans to expand the program to children and adolescents up to 18 years old.

Dravet syndrome is a rare and severe form of epilepsy that usually begins during infancy. Patients can experience frequent seizures as well as developmental and other neurological problems. The disease is often linked to changes in the SCN1A gene.

Encoded said recent interim Phase 1/2 POLARIS data showed substantial and sustained reductions in seizure frequency in patients treated with ETX101, along with encouraging signs of developmental improvement.

The company plans to use part of the new financing to strengthen its manufacturing capabilities. This includes scaling up its internal Good Manufacturing Practice (GMP) manufacturing operations to support the future development and potential commercialisation of its therapies.

The funding will also help advance Encoded’s wider pipeline. The company expects to move ETX301 toward an Investigational New Drug (IND) submission in 2027 for the treatment of post-amputation neuroma pain.

Kartik Ramamoorthi, Ph.D., CEO of Encoded Therapeutics, said the recent clinical data increased the company’s confidence in ETX101 and its potential to change the course of Dravet syndrome. He added that the financing will provide resources to move the program through pivotal development while expanding Encoded’s broader platform and pipeline.

GV partners also highlighted the progress of ETX101, pointing to its sustained seizure control and early neurodevelopmental signals. GV said the results support Encoded’s approach to developing more precise and durable genetic medicines for neurological diseases.

With the new financing, Encoded plans to focus on the pivotal development of ETX101 while continuing to build its manufacturing infrastructure and advance additional genetic medicine candidates.

The company’s latest funding round gives it significant financial support as it moves closer to potentially bringing a new treatment option for patients with Dravet syndrome into late-stage development.