The company’s approach focuses on identifying genetically validated targets with relevance across both rare and common diseases, aiming to advance precision therapies in areas of high unmet medical need.

The company’s lead program, ABS-1230, is an oral KCNT1 inhibitor in development for KCNT1-related epilepsy, a rare pediatric condition with no approved treatments. The program has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration. A Phase I trial in healthy volunteers is expected to begin in late 2025, with a Phase Ib study in patients planned for early 2026.

In June 2025, Actio secured $66 million in Series B financing co-led by Regeneron Ventures and Deerfield Management, with participation from Canaan, Droia Ventures, and Euclidean Capital. The funding will support advancement of both clinical programs and continued pipeline expansion.