GBT440, our lead drug candidate, is being developed as an oral, once-daily therapy for patients with sickle cell disease (SCD). We believe GBT440 holds significant potential for SCD patients and are investigating the compound as a mechanism-based and potentially disease-modifying therapeutic for this grievous disease.
In our ongoing clinical development program, we are evaluating the impact of GBT440 on abnormal polymerization of deoxy-hemoglobin, the underlying mechanism of red blood cell sickling. The ability to address SCD at this fundamental level of pathology provides the potential to directly treat the disease rather than only its symptoms. We believe that this mechanism is relevant for all patients with SCD and have designed a clinical program targeted at the treatment of adult and pediatric SCD patients across all genotypes.
Currently, GBT440 is being investigated in an ongoing Phase 1/2 clinical trial in both healthy subjects and SCD patients.