NEW YORK – Actio Biosciences on Tuesday said it has raised $55 million in Series A financing that it hopes to use to advance precision medicines for rare and common diseases.
Canaan and DROIA Ventures led the funding round; existing investors Deerfield Management and EcoR1 also contributed funds, as did new investor Euclidean Capital.
Actio has a human genetics platform that includes a database, called the Rare Disease Target Atlas. The San Diego-based company's experts in bioinformatics, biology, and chemistry use the platform to identify and understand the function of relevant disease targets in rare disease and create drugs that modulate those targets. The firm wants to leverage its understanding of the role of these targets in rare indications to eventually develop treatments against those same targets for common diseases.
Actio is collaborating with the Jackson Laboratory's Rare Disease Translational Center and using its genetically engineered mouse models to evaluate drug targets and phenotypes of interest. Actio is also working with other academic centers to validate the role of certain genes in disease, garner deeper insights into disease pathology, investigate the natural history of rare diseases, and, in the future, conduct clinical trials.
The lead program in Actio's pipeline is a drug targeting TRPV4, which it plans to first evaluate as a potential treatment for rare diseases caused by TRPV4 mutations, including Charcot-Marie-Tooth disease type 2C. CMT2C causes nerve damage, mobility issues, foot deformities, and respiratory complications. Based on preclinical studies, Actio believes this agent may improve motor function, mobility, bone morphology, and possibly increase the lifespan of those with CMT2C.
"Since our founding [in 2021], we've built a proprietary platform designed to de-risk targets of interest and establish biological connections between rare and common disease states that is unlike anything else in the industry today," John McHutchison, cofounder and an Actio board member, said in a statement. "That work has led to the prioritization of several high-value targets, including our lead TRVP4 program for the treatment of CMT2C and other bone diseases with high unmet need."
McHutchison added that the company is working to expand its drug pipeline and has flagged two programs that show promise in epilepsies and skin conditions caused by genetic abnormalities.