SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Encoded Therapeutics Inc., a biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, today highlights oral and poster presentations on the Company’s preclinical gene therapy programs and vector engineering platform at the 27th ASGCT Annual Meeting being held May 7 – 11, 2024 in Baltimore, MD.“ We are thrilled to share multiple advancements across our portfolio of precision genetic medicines, showcasing substantial progress across various fronts. These data underscore our platform’s potential to deliver precision therapies for a range of CNS diseases,” said Stephanie Tagliatela, Chief Scientific Officer at Encoded. “ Alongside clinical advancement of our lead program, ETX101 for SCN1A+ Dravet syndrome, we are poised to nominate multiple programs to development in 2025, marking yet another significant milestone.”Oral PresentationTitle: GABA Selective AAV-mediated Gene Therapy Provides Durable Seizure Protection in Multiple Refractory Epilepsy Models (Abstract #19)Session: AAV Vectors - Preclinical and Proof-of-Concept: Therapy FocusDate & Time: Tuesday, May 7, 1:45–2:00 p.m. ETLocation: Ballroom 2Epilepsy affects approximately 1% of the population, with nearly one third of cases failing to respond to first- and second-line treatments. Encoded’s vector engineering platform has enabled the development of GABA-selective AAV-mediated gene therapies designed to potentiate GABAergic neurotransmission and modulate the circuit dysfunction underlying refractory epilepsies. Our lead candidate reduced seizure incidence and severity in two biologically distinct seizure assays with no adverse impact on body weight, motor function or locomotion

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--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 ETX101 as a potential treatment for children with SCN1A+ Dravet syndrome. Dravet syndrome is the most common developmental and epileptic encephalopathy and is characterized by a spectrum of clinical symptoms, including treatment-resistant seizures and neurodevelopmental stagnation. The UK CTA for EXPEDITION is part of a global regulatory strategy established by Encoded for clinical development of ETX101, which also includes regulatory clearance for an investigational new drug (IND) application in the United States and approval under the Clinical Trial Approval (CTA) scheme in Australia. “This CTA marks the third regulatory approval for ETX101 as we advance our global clinical development program, POLARIS, to bring this potential one-time AAV gene regulation therapy to people affected by SCN1A+ Dravet syndrome around the world,” said Sal Rico, M.D., Ph.D., Chief Medical Officer of Encoded. “We believe ETX101 has the potential to deliver transformative benefit in multiple disease domains and have designed our clinical program to efficiently assess its impact on affected infants and young children. We are committed to working closely with our highly experienced investigators to expeditiously enroll affected children in an effort to advance therapeutic options for people living with Dravet syndrome.”

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--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 developmental and epileptic encephalopathy, experience a spectrum of clinical symptoms, including treatment-resistant seizures and neurodevelopmental stagnation. Dravet syndrome is primarily caused by loss-of-function variants in the SCN1A gene, accounting for over 85% of cases. ETX101 is an AAV9-mediated candidate gene regulation therapy designed to selectively upregulate expression of the SCN1A gene in GABAergic inhibitory interneurons and potentially address the underlying cause of the disease. ETX101 Global Clinical Development. Encoded has received clearance for its Investigational New Drug (IND) application from the US Food and Drug Administration and approval under the Clinical Trial Approval (CTA) scheme from the Australia Therapeutic Goods Administration to initiate clinical trials of its gene therapy candidate, ETX101