Our founders discovered that certain short chain fatty acids (SCFAs) had the potential to be used as powerful therapeutic agents for several serious and life-threatening hematologic disorders. They identified novel epigenetic mechanisms of action in which these compounds target the expression of key genes required to generate their therapeutic effects. Several of these SCFA compounds were evaluated in the laboratory as well as relevant pre-clinical animal models. HemaQuest has selected one of these compounds, HQK-1001, for clinical development to treat hemoglobin disorders. HQK-1001 has demonstrated favorable safety profiles both in animal studies as well as early clinical studies in healthy volunteers and in patients with hemoglobin diseases. HQK-1001 has shown promising pharmacodynamic and therapeutic effects in early stage clinical trials in sickle cell disease and beta thalassemia. HemaQuest is currently advancing HQK-1001 in a Phase 2b clinical trial in sickle cell disease, which if successful, could lead to pivotal trials in 2015.