Our approach
science
Unique Genetics Approach to Maximize Clinical Benefit for Patients
Actio builds on decades of progress in human genetics to identify the genetic causes of disease and turn those insights into medicines. We start by studying rare genetic diseases with high unmet need, using advanced data tools to identify the right biological targets and understand how specific mutations drive disease. We then design precision small molecule medicines that can address all disease-causing variants of a target, with the goal of helping every patient. Finally, we apply what we learn to expand these treatments into more common diseases that share the same underlying biology.
Our approach is
built on four
foundational pillars
Target identification
Conduct rigorous target selection by evaluating the universe of genes related to rare Mendelian diseases and applying our advanced informatics tools and deep genetics expertise to develop hypotheses of more prevalent indications with related underlying biology
Molecule design
Design potent small molecule modulators to address all disease-causing variants of the identified target to enable development of inclusive treatments for all patients with the rare disease
Mutation characterization
Characterize the biology of pathogenic Mendelian mutations in a target gene and leverage translatable preclinical models to further understand underlying disease mechanisms and determine the direction of therapeutic modulation required
Indication expansion
Confirm our hypotheses of additional more prevalent indications by applying learnings from clinical and preclinical data as well as high-throughput machine learning algorithms that analyze population genetics
We used this approach to advance our three programs and intend to add new programs to our pipeline by leveraging this repeatable approach.
