Our science and platform
science
Unique Genetics Approach to Maximize Clinical Benefit for Patients
Actio develops transformational medicines for rare diseases with high unmet need by precisely targeting underlying causes of rare disease. We are pioneering a genetic framework to match our rare disease targets to more common indications with shared underlying biology.
Ensure Every Patient is Treated
- Conduct functional analysis of pathogenic mutations
- Build disease relevant assays
- Confirm direction of modulation
- Deep pathway understanding
Definitive Preclinical Models
- Develop construct-valid preclinical models
- Identify relevant biomarkers
- Test the therapeutic hypotheses and potential to reverse disease
Feasible Rare Disease Trials
- Leverage natural history studies to define endpoints and biomarkers
- Conduct first interventional trials in de-risked homogenous diseases
Concrete Biological Pointers to Expansion Opportunities
- Identify the most appropriate diseases
- Leverage learnings from monogenic rare disease
- Expand the treatable patient population
Proprietary platform to translate genetic insights
While it is well known that a single gene can lead to a rare disease, the function of mutations in those single genes and how they might be modulated for therapeutic intervention is often not well characterized. 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. Actio’s platform comprises a vast proprietary target database, called the Rare Disease Target Atlas, and unparalleled expertise in bioinformatics, biology and chemistry.
We partner with industry leaders to provide validation for our targets and to help define clinical endpoints
Our partnership with the Rare Disease Translational Center at The Jackson Laboratory (JAX) is focused on evaluating targets and phenotypes of interest in high-quality genetically engineered mouse models at scale, enabling a high level of confidence in the outcomes observed.
Through collaborations with prominent academic centers, we gain 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.
