Science

Our Approach

GBT is committed to transforming the treatment of severe blood-based disorders. Our scientific focus and mission is to develop novel medicines that treat grievous blood-based disorders by arresting the underlying chemical and biological mechanisms of the disease.

 

As one of the largest and most accessible organs, blood is a rich source of biologically and clinically validated targets. These targets can involve cellular components in the blood, including red blood cells (RBCs), platelets and white blood cells, as well as proteins, including proteases, circulating in plasma that are critical for homeostatic balance for a number of biological pathways, including the process of blood coagulation.

At GBT, our research activities focus on well characterized and validated blood-based targets that have not yet been successfully drugged. We apply our knowledge of structural biology-based drug design and medicinal chemistry to discover and optimize compounds for their activity against these targets, supported by strong clinical hypotheses and a clear path to clinical proof-of-concept.

 

Our efforts have produced a pipeline of novel, mechanism-based small molecule therapeutics that we believe have the potential to address the underlying chemical and biological mechanisms of various blood-based disorders. Using this approach, we were able to successfully advance GBT440, our lead product candidate, from molecular modeling into clinical development in less than three years.