Treating the Root Cause of Sickle Cell Disease

RARECast

The genetic basis of sickle cell disease has been long understood, but it has only been recent that new treatments and a growing pipeline of therapies have emerged. The inherited blood disorder causes red blood cells to become crescent shaped, which restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues leading to severe pain and organ damage. At the end of 2019, Global Blood Therapeutics won accelerated approval for Oxbryta, the first FDA-approved therapy that directly inhibits sickle hemoglobin polymerization, the root cause of sickle cell disease. We spoke to Ted Love, president and CEO of Global Blood Therapeutics, about the changing landscape of sickle cell disease, Oxbryta and the company’s pipeline behind it, and how the company is approaching the global need for the drug given the higher prevalence of the disease in parts of the world.