Actio Biosciences Raises $66 Million to Advance Genetics-Driven Pipeline

Rare Daily Staff

Actio Biosciences, a clinical-stage biotechnology company leveraging a novel approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, has raised $66 million in series B financing.

New investor Regeneron Ventures and current investor Deerfield Management co-led the financing, with participation from existing investors Canaan, Droia Ventures, and Euclidean Capital.

The financing will support the advancement of Actio’s lead programs: ABS-1230, a KCNT1 inhibitor for the treatment of KCNT1-related epilepsy and other genetic epilepsies; and ABS-0871, a TRPV4 inhibitor currently being evaluated in a Phase 1 healthy volunteer clinical trial for the treatment of Charcot-Marie-Tooth disease type 2C (CMT2C) and overactive bladder. CMT2C is a rare inherited disorder that affects both motor and sensory functions.

In preclinical studies, ABS-1230 has been shown to inhibit all recurrently observed pathogenic mutations in the KCNT1 gene, making it broadly applicable to patients with KCNT1-positive epilepsy. Actio plans to initiate the healthy volunteer portion of a phase 1 clinical trial of ABS-1230 in the second half of 2025, with plans to expand into a proof-of-concept phase 1b study in KCNT1-related epilepsy patients in early 2026. The U.S. Food and Drug Administration recently granted ABS-1230 both Rare Pediatric Disease designation and Orphan Drug designation.

ABS-0871 is a potential first-in-class oral, small-molecule TRPV4 inhibitor for the treatment of CMT2C. Preclinical evaluation of the program in novel, construct-valid CMT2C rare disease models have demonstrated marked improvements in motor function and mobility compared to untreated controls. Given its mechanism of action targeting TRPV4, ABS-0871 may also have therapeutic potential for the treatment of overactive bladder. Actio is currently advancing ABS-0871 in the healthy volunteer portion of a phase 1 clinical trial, with plans to expand into a phase 1b study in patients with TRPV4+ CMT2C in 2026. The FDA granted ABS-0871 Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation.

“We have made tremendous progress across our pipeline—executing a precision medicine strategy that targets the root causes of disease through genetically informed drug development,” said David Goldstein, co-founder and CEO of Actio. “ABS-1230 and ABS-0871 have the potential to be transformative, disease-modifying therapies in their respective rare indications, and growing evidence supports expansion into broader indications.”

Photo: David Goldstein, co-founder and CEO of Actio