SOUTH SAN FRANCISCO, Calif. — Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today announced the approval of its Clinical Trial Authorisation (CTA) application by the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA) for the EXPEDITION Study of...
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SOUTH SAN FRANCISCO, Calif. — Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe neurological disorders, today announced the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ETX101 following review of preliminary seizure data from patients treated in its ongoing...
SOUTH SAN FRANCISCO, Calif. — 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...
SOUTH SAN FRANCISCO, Calif. — Encoded Therapeutics Inc., a biotechnology company focused on developing genetic medicines for severe central nervous system (CNS) disorders, today outlined the global development strategy for its lead gene therapy candidate, ETX101, for the treatment of SCN1A+ Dravet syndrome. Individuals with Dravet syndrome, the most common...
SOUTH SAN FRANCISCO, Calif. — 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...
SOUTH SAN FRANCISCO, Calif. — 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+...
SOUTH SAN FRANCISCO, Calif. — 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...
SOUTH SAN FRANCISCO, Calif. — Encoded Therapeutics Inc., a clinical-stage genetic medicines company, today announced upcoming presentations of interim clinical data from its ongoing POLARIS Phase 1/2 open-label trials evaluating ETX101 in children with SCN1A+ Dravet syndrome. The data will be featured at the American Epilepsy Society Annual Meeting, taking place...
The foundation End AxD, a nonprofit dedicated to research, treatment and a cure for Alexander disease, has donated $74,000 to UMass Chan Medical School to explore a gene therapy treatment for the fatal neurological disease. The funding will go to Jun Xie, PhD, associate professor of microbiology & physiological systems,...
SAN DIEGO – Endeavor BioMedicines, a clinical-stage precision medicine company targeting the core drivers of multiple terminal diseases including oncology and fibrosis, announced that the company has dosed its first patient in a Phase 2 trial of taladegib (ENV-101) to treat idiopathic pulmonary fibrosis (IPF). Taladegib is a small-molecule inhibitor...