Actio Biosciences is a clinical-stage company using genetics and precision medicine to develop oral small-molecule therapeutics that target shared underlying biology across rare and common diseases. Its pipeline is led by ABS-1230, a selective KCNT1 inhibitor for KCNT1-related epilepsy, and ABS-0871, a TRPV4 inhibitor being evaluated for Charcot-Marie-Tooth disease type 2C and overactive bladder. ABS-0871 is currently in the healthy volunteer portion of a Phase 1 trial with plans to expand into a Phase 1b patient study in 2026; ABS-1230 is planned to enter the healthy volunteer portion of a Phase 1 trial in the second half of 2025 with a Phase 1b proof-of-concept study in KCNT1-related epilepsy patients planned for early 2026. ABS-1230 has received rare pediatric and orphan drug designations from the FDA; ABS-0871 has rare pediatric, orphan drug and fast track designations. The company applies genetics, drug discovery and data sciences to select programs with reduced biological and technical risk, aiming to streamline development of precisely targeted therapies. Founded in October 2021 and based in San Diego, Actio positions its programs for expansion into broader indications as supporting evidence emerges. Actio Biosciences applies a human genetics platform to identify and de-risk therapeutic targets, aiming to translate insights from rare diseases into treatments for common conditions. Its platform includes a proprietary Rare Disease Target Atlas and expertise in bioinformatics, biology and chemistry. The company has a partnership with The Jackson Laboratory to evaluate targets in genetically engineered mouse models and collaborates with academic centers for further validation and natural history insights. Actio’s lead program targets the TRPV4 ion channel and is being advanced toward the clinic for TRPV4-related rare diseases such as CMT2C and serious bone disorders; preclinical models showed improvements in motor function, mobility, bone morphology and lifespan. The company plans to expand its pipeline and has identified two additional programs aimed at genetic epilepsies and genetic skin conditions. Actio completed a $55 million Series A to advance its programs and platform.

Total raised

$121M

Funding rounds

2

Latest round

Series B

Latest activity

Jun 2025