Rare epilepsy specialist Actio secures $66 million in series B

19 June 2025

US biotech Actio Biosciences has raised $66 million in a series B round to support its pipeline of genetically targeted therapies, with new funding led by Regeneron Ventures and repeat backing from Deerfield Management.

The San Diego-based company is developing oral, first-in-class small molecules aimed at serious rare diseases with genetic drivers—namely KCNT1-related epilepsy and Charcot-Marie-Tooth disease type 2C (CMT2C). Additional investors included Canaan, Droia Ventures and Euclidean Capital.

The funds will support continued development of Actio’s lead candidates: ABS-1230, a KCNT1 inhibitor slated to enter a Phase I trial later this year, and ABS-0871, a TRPV4 inhibitor already in early-stage testing. Both programs target ultra-rare conditions that affect around 2,500 individuals each in the USA.

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