Beta-thalassemia is a rare, genetic blood disease that can result in severe anemia and lifelong dependence on red blood cell transfusions, a lengthy process that patients typically undergo every 2 to 5 weeks.Zynteglo works by adding functional copies of a modified form of the beta-globin gene...
If Moderna Therapeutics is the belle of the biotech ball right now, then Noubar Afeyan is one of her proud papas. Afeyan co-founded the company with esteemed drug delivery guru Robert Langer in 2011 out of his Flagship VentureLabs, the arm of Flagship Ventures that creates and launches start...
Most crises eventually abate on their own, as the sickle cells break down and blood flow improves, bringing new oxygen. Severe crises require morphine for relief or even hospitalization, often causing children to miss days and sometimes weeks of school. Each crisis, no matter where it localizes...