Articles from Encoded Therapeutics Inc.

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 December 5–9, 2025, in Atlanta, Georgia.

Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe neurological disorders, today announced preclinical data demonstrating that its AAV9 microRNA (miRNA) gene therapy candidate achieved robust knockdown of SCN9A (NaV1.7), a key mediator of pain signaling, in non-human primates (NHPs). The levels of target engagement in NHPs exceeded therapeutic thresholds observed in an established rodent pain model. Together, these findings support the potential of Encoded’s candidate as a one-time, non-opioid gene therapy for intractable chronic pain.

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 Phase 1/2 program. ETX101 is an AAV9-based gene regulation therapy designed to increase SCN1A expression in GABAergic inhibitory neurons for the treatment of SCN1A+ Dravet syndrome. ETX101 has Fast Track, Rare Pediatric, and Orphan Drug Designations from FDA and Orphan Designation from EMA.

Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, today announced the results of preclinical studies that underscore the strength of the Company’s vector engineering platform and demonstrate continued progress of programs for Angelman syndrome, Alzheimer’s disease / tauopathies and chronic pain. Three poster presentations are being highlighted at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, LA, May 13 – 17, 2025.

Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, today announced three presentations that underscore the strength of the Company’s vector engineering platform and demonstrate continued progress of the ETX201 in Angelman syndrome (AS), chronic pain and Alzheimer’s disease / tauopathies programs. The presentations will be made at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, LA, May 13 – 17, 2025.

Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, today announced continued progress in its lead clinical program, ETX101, and highlighted corporate achievements that advance its research pipeline and further develop infrastructure to fully integrate the Company’s gene therapy capabilities.

Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, announced today that it will present new preclinical data on ETX201, an AAV9-based vectorized miRNA approach designed to reduce the expression of the UBE3A-ATS and unsilence paternal UBE3A expression for the treatment of Angelman syndrome.

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.

Encoded Therapeutics Inc., a biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, today unveiled multiple pipeline programs ahead of the 27th ASGCT Annual meeting on May 7 - 11, 2024 in Baltimore, MD. Presentations will highlight progress of preclinical programs for Angelman syndrome, Lennox-Gastaut syndrome (LGS), STXBP1-related disorders (STXBP1-RD), Alzheimer’s disease (MAPT) and neuropathic pain (SCN9A), together with innovations in the company’s vector engineering platform.

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.

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.