Actio Biosciences Raises $55 Million to Advance Precision Medicine Approach for Rare and Common Diseases

Rare Daily Staff

Actio Biosciences raised $55 million in a series A financing to advance a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases.

Canaan and DROIA Ventures led the financing, with participation from existing investors Deerfield Management and EcoR1, as well as new investor Euclidean Capital.

“Defining a high-impact drug target for a common disease with a heterogenous population has a very low probability of success and has long been a major challenge for drug discovery,” said David Goldstein, co-founder and CEO of Actio. “By starting with a focus on rare diseases, we gain invaluable insights that not only define new therapeutic benefits in high-need indications but also de-risk and guide therapeutic expansion to more common conditions, benefiting even more patients.”

While it is well known that a single gene can lead to rare disease, the function of mutations in those single genes and how they might be modulated for therapeutic intervention is often not well understood. To overcome this and better utilize the breadth of genetic information now available for drug discovery and development, Actio is leveraging its human genetics platform to identify relevant disease targets, understand their function in disease and create medicines that modulate them.

The company’s platform comprises a vast proprietary target database, called the Rare Disease Target Atlas, and expertise in bioinformatics, biology, and chemistry. Actio will initially focus on identifying targets and advancing programs for rare disease indications and plans to leverage insights throughout the development process to inform potential relevance of those targets in more common diseases.

The company has established a partnership with the Rare Disease Translational Center at The Jackson Laboratory, a leading nonprofit biomedical research institution dedicated to understanding the molecular underpinning of rare diseases and their treatments, to evaluate targets and phenotypes of interest in high-quality genetically engineered mouse models at scale and enabling a high level of confidence in the outcomes observed. Actio is also collaborating with prominent academic centers to provide further validation of the role of certain genes in disease, as well as insights into disease pathology to better inform the design of preclinical studies, rare patient natural history studies and future clinical trials.

Actio’s lead program targets TRPV4, an ion channel target identified as high-value through the company’s platform. Actio plans to initially evaluate it for the treatment of rare diseases caused by TRPV4 mutations, including Charcot-Marie-Tooth disease type 2C, characterized by severe muscle weakness, vocal cord paresis and respiratory complications, as well as serious bone diseases, such as metatropic dysplasia.

Evaluation of this program in novel construct-valid preclinical TRPV4 rare disease models has demonstrated marked improvements in motor function, mobility and bone morphology, as well as increased lifespan compared to untreated controls. Beyond this program, Actio is working to expand its pipeline and has identified two additional programs that it plans to evaluate for genetic epilepsies and genetic skin conditions.

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