Encoded Therapeutics – Translating Next-Generation Science into . . . Our novel targeting and regulation platform enables us to target any cell type that has a unique genetic profile Encoded product candidates are designed to overcome first-generation gene therapy challenges by conferring potentially increased gene specificity, cell selectivity and or potency
News – Encoded Therapeutics Encoded Therapeutics to Present New Data for CNS Gene Therapy Programs at the ASGCT 28th Annual Meeting February 13, 2025 Encoded Therapeutics Reports Clinical Progress of ETX101 Gene Therapy for Dravet Syndrome, Recaps 2024 Corporate Achievements and Provides 2025 Outlook
Programs – Encoded Therapeutics Encoded's cell-selective gene therapy approach enables us to tap into the diversity of different cell types in the brain Programs Encoded is building an internally developed pipeline of best- and potentially first-in-class therapies focused on central nervous system (CNS) disorders
ETX101 for Dravet Syndrome – Encoded Therapeutics Encoded is developing ETX101, a potential one-time, disease-modifying gene regulation therapy for SCN1A+ Dravet syndrome ETX101, Encoded’s lead program, is specifically designed to address the underlying cause of Dravet syndrome, the most common developmental and epileptic encephalopathy
Encoded Therapeutics Reports Clinical Progress of ETX101 Gene Therapy . . . Encoded’s second program, ETX201, is a development-stage vectorized miRNA-based gene therapy designed to restore expression of UBE3A in individuals with Angelman syndrome In parallel, we are advancing potentially best-in-class programs for common CNS conditions, including chronic pain and Alzheimer’s disease
Our Science – Encoded Therapeutics Encoded engineers these regulatory elements to precisely target specific cell types relevant to disease Using genomics-driven screening methods, we screen thousands of naturally-occurring regulatory sequences in the human genome that drive robust and precise gene expression
Pipeline – Encoded Therapeutics Our lead program is for Dravet syndrome, which is a severe, developmental and epileptic encephalopathy characterized by frequent, treatment-resistant and prolonged seizures, significant developmental delays, movement and balance issues and sleeping and behavioral difficulties
About – Encoded Therapeutics We are fully equipped and well-positioned to realize the potential of Encoded’s novel approach to gene therapy for people living with central nervous system (CNS) diseases and their families
Encoded Therapeutics Announces US IND Clearance and Australian CTA . . . 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
Encoded Therapeutics Presents Promising New Data for CNS Gene Therapy . . . New data in chronic pain include the first demonstration of preclinical efficacy for Encoded’s SCN9A-targeted vectorized miRNA candidates in a rat pain model, showing a durable analgesic effect and up to 70 percent Scn9a knockdown in DRG tissue