Actio Biosciences Secures $66 Million Series B Financing to Advance Genetics-Driven Pipeline of Small Molecule Therapeutics for Rare and Common Diseases

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, today announced the closing of a $66 million 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.

Actio’s pipeline is led by 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.

“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, Ph.D., 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. This new funding from industry-leading investors speaks to the value of our approach and provides us with important resources to continue advancing our programs.”

The Series B financing will support the advancement of Actio’s lead programs, ABS-1230 and ABS-0871.

ABS-1230 is a potential first-in-class oral, selective small molecule KCNT1 inhibitor for the treatment of KCNT1-related epilepsy, a rare and often fatal pediatric epileptic encephalopathy with a United States (U.S.) prevalence of approximately 2,500 individuals. In preclinical studies, ABS-1230 has been shown to inhibit all recurrently observed pathogenic mutations in the KCNT1 gene, making ABS-1230 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. ABS-1230 was recently granted both rare pediatric and orphan drug designations from the U.S. Food and Drug Administration (FDA).

ABS-0871 is a potential first-in-class oral, small molecule TRPV4 inhibitor for the treatment of CMT2C, a rare inherited disorder that affects both motor and sensory functions with a U.S. prevalence of approximately 2,500 individuals. Preclinical evaluation of this program in novel construct valid CMT2C rare disease models has 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. ABS-0871 was granted rare pediatric drug, orphan drug and fast track designations from the FDA.

“Actio has done an exceptional job of identifying serious unmet needs in rare disease and advancing solutions that may also be applicable to patients with more common diseases,” said Jason Fuller, Ph.D., Partner, Deerfield Management. “We’re pleased to support the company, encouraged by its progress, and excited by the team’s potential to make a difference for patients.”

About Actio Biosciences

Actio Biosciences is a clinical-stage company leveraging advances in precision medicine to develop new therapeutics that target shared genetics in rare and common diseases—bringing meaningful medicines from one to many. Applying its expertise in genetics, drug discovery and data sciences, Actio seeks to identify programs where both biological and technical risk can be minimized to streamline the drug development process and bring forward exceptionally potent and precisely targeted therapeutics. Actio is advancing two lead rare disease programs – ABS-0871 and ABS-1230 – with first-in-class potential for the treatment of Charcot-Marie-Tooth disease, type 2C (CMT2C) and KCNT1-related epilepsy, respectively. Founded in October 2021, the San Diego-based company is guided by leaders in genetics and drug development and backed by top healthcare investors. For more information, please visit ActioBiosciences.com and follow the company on LinkedIn and X.

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