Articles from Encoded Therapeutics, Inc.

Encoded Therapeutics, Inc. (“Encoded”), a clinical-stage biotechnology company developing precision genetic medicines for severe neurological disorders, today announced that new Phase 1/2 POLARIS data for ETX101, its investigational AAV9-based gene regulation therapy designed as a one-time, disease-modifying treatment for SCN1A+ Dravet syndrome, were presented in an oral session at the 16th European Epilepsy Congress (EEC) in Athens, Greece by Professor Ingrid Scheffer, Pediatric Neurologist and Laureate Professor of Pediatric Neurology at The University of Melbourne.

Encoded Therapeutics, Inc. (“Encoded”), a clinical‑stage biotechnology company developing precision genetic medicines for severe neurological disorders, today will present an expanded dataset from the ongoing POLARIS Phase 1/2 trials of ETX101, its investigational AAV9‑based gene regulation therapy designed as a one‑time, disease‑modifying treatment for SCN1A+ Dravet syndrome. The update includes additional patients, early data from the top dose level (DL4), and longer‑term outcomes that further define the emerging clinical profile of ETX101 in children aged 6 months to 7 years. These results will be featured in an oral presentation during the Presidential Symposium (Wednesday, May 13, 2:28–2:39 p.m. ET) at the 29th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT).

Encoded Therapeutics, Inc. (“Encoded”), a clinical-stage biotechnology company developing precision genetic medicines for severe neurological disorders, today announced that the first patient has been dosed in the pivotal ENDEAVOR Part 2 study of ETX101 for Dravet syndrome, marking an important transition into late-stage clinical development for the program. Encoded has also initiated dosing in the ENDEAVOR Part 1B expansion study in patients aged 4 to 18 years, extending evaluation of ETX101 across a broader patient population.

Encoded Therapeutics Inc., a clinical-stage genetic medicines company, today announced its participation in the 29th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place May 11–15, 2026, in Boston, Massachusetts. Encoded will present three abstracts, including one oral presentation in the Presidential Symposium and two scientific posters, demonstrating the breadth of its vector engineering platform across severe neurological disorders.

Encoded Therapeutics, Inc. (“Encoded”), a clinical-stage biotechnology company developing precision genetic medicines for severe neurological disorders, today announced the successful completion of its Initial Comprehensive Multidisciplinary Regenerative Medicine Advanced Therapy (RMAT) meeting with the U.S. Food and Drug Administration (FDA), and alignment on the pivotal study design supporting the company’s planned BLA submission for ETX101, an AAV9-based gene regulation therapy designed as a one-time, disease-modifying treatment for SCN1A+ Dravet syndrome.

Encoded Therapeutics, Inc. (“Encoded”), a clinical-stage biotechnology company developing precision genetic medicines for severe neurological disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to ETX101 for the treatment of SCN1A+ Dravet syndrome. This designation is in addition to Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease Designations, all previously granted by the FDA.