Targeting the Regulatory Genome to Treat Rare Blood Diseases

Syros Pharmaceuticals is developing a platform for targeting the regulatory genome with therapies to address diseases at a fundamental level. At the end of last year, the company entered into a collaboration with Global Blood Therapeutics to develop new therapies for sickle cell disease and beta thalassemia. Under the collaboration, Syros is using its platform to discover drugs that turn on the production of fetal hemoglobin as a way to treat these rare, hereditary, blood disorders. The production of fetal hemoglobin is usually shut down soon after birth. We spoke to David Roth, chief medical officer of Syros Pharmaceuticals, about the collaboration, how Syros’ approach works, and why activating fetal hemoglobin is viewed as a promising approach to treating these genetic blood diseases.sickle cell diseasebeta thalassemiagenetic disease