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Actio Biosciences is advancing two first-in-class, orally available small molecule inhibitors: ABS-0871 (TRPV4 inhibitor) in Phase 1 for CMT2C/overactive bladder and ABS-1230 (KCNT1 inhibitor) entering Phase 1 in 2025 for KCNT1-related epilepsies. Both have received orphan/rare pediatric drug designations from FDA; preclinical models show marked benefit, and clinical results pending.
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