Global Blood Therapeutics (GBT) and Syros Pharma announced yesterday their partnership to discover, develop and commercialize novel therapies for SickleCell Disease (SCD) and β-thalassaemia. Under the agreement, Syros will use its leading gene control platform to identify therapeutic targets and discover drugs that induce fetal hemoglobin, and GBT will receive an option to obtain an exclusive worldwide license to develop, manufacture and commercialize products resulting from the collaboration.
“We believe that Syros’ approach to inducing fetal #haemoglobin is one of the most promising ways to identify the next generation of therapies to treat sickle cell disease and beta thalassaemia at a fundamental level We will continue to seek the best scientific approaches to transform the treatment of these devastating lifelong diseases”, stated Ted W. Love, M.D., president and CEO of GBT.
Using its gene control platform to elucidate mechanisms controlling gamma globin gene expression, Syros identified components of LRF (leukemia/lymphoma-related factor) and the NuRD (nucleosome remodeling and histone deacetylation) complex that could serve as potential targets to switch on the gamma globin gene, which is normally silenced a few months after birth. By turning on gamma globin expression, GBT and Syros aim to induce the production of fetal hemoglobin, which is known to exert protective effects on the red blood cells of patients with SCD and beta thalassaemia and mitigate the clinical manifestation of these diseases.