ETX101
Dravet Syndrome
Phase 3Active
Key Facts
About Encoded Therapeutics
Encoded Therapeutics is a private, pre-revenue biotech focused on next-generation gene therapies for neurological diseases. Its core differentiator is a genomics-driven platform that engineers regulatory elements (promoters, enhancers) to achieve cell-type-specific expression of therapeutic transgenes, aiming to overcome the off-target toxicity and limited specificity of first-generation AAV vectors. The company's lead program, ETX101 for Dravet syndrome, has received FDA Breakthrough Therapy Designation and is advancing into a pivotal study, positioning Encoded as a notable player in the precision neurology space.
View full company profileTherapeutic Areas
Other Dravet Syndrome Drugs
| Drug | Company | Phase |
|---|---|---|
| BL-001 | Bloom Science | Phase 2 (Planned) |
| Cannabidiol Program | Nexien BioPharma | Pre-clinical |
| RT101 | Regel Therapeutics | Preclinical |
| Zorevunersen (STK-001) | Stoke Therapeutics | Phase 3 |
| EPX-100 (clemizole) | Harmony Biosciences | Phase 1/2 |