Gene Therapy Pipeline for SCD and Thalassemia—Cost and Access Become Key Under Kennedy HHS

Sickle cell disease (SCD) and beta-thalassemia are hereditary blood disorders caused by mutations in the β-globin gene, leading to abnormal hemoglobin and debilitating health issues. SCD afflicts millions globally (about 100,000 in the U.S. and ~50,000 in Europe), causing episodic vaso-occlusive pain crises, organ damage, infections, and early mortality. Beta-thalassemia, a related hemoglobin disorder, affects fewer patients (on the order of thousands in the U.S. and tens of thousands in Europe) but similarly requires lifelong medical management (e.g., frequent blood transfusions). Until recently, the only cure for either disease was a bone marrow transplant from a matched donor, a risky option available to only a fraction of patients. Today, however, an unfolding pipeline of gene therapies promises functional cures by fixing or replacing the defective gene. In late 2023, the FDA approved the first two gene therapies for SCD, marking a historic breakthrough. Yet the roll-out of these “cures” has exposed major challenges – sky-high costs, access barriers, and questions of equity – that investors and innovators are now racing to address…

View the rest of the article here

View the PDF