Bringing Meaningful Therapies From One To
Translating genetic insights into novel precision medicines
At Actio, we are leveraging advances in genetics and precision medicine to develop life-changing treatments that target underlying shared biology between rare and common diseases. We are applying deep expertise in Mendelian genetics, drug discovery, and data science to pinpoint targets that not only cause severe rare diseases with high unmet need, but that also hold promise in more prevalent indications.
Decoding Genetic Insights
A deep understanding of human genetics is core to our approach. 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. By starting with a focus on rare diseases – where we’re looking at a defined population and know that one gene is leading to one disease – we aim to enhance our understanding of both the target biology and disease. Through this approach, we gain invaluable insights that can de-risk and guide therapeutic expansion to more prevalent diseases.
TEAM
We believe that advancing new treatments for diseases that affect one patient is fundamentally as important as advancing new treatments for diseases that affect many.
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