Gene-based therapies show promise in transforming sickle cell disease.
Two gene-based treatments for sickle cell disease, a genetic blood disorder that mostly affects people of color, could be approved by the FDA late this year or early in 2024. The treatments, a gene therapy from Bluebird Bio and a gene-editing treatment by a partnership between Vertex Pharmaceuticals and CRISPR Therapeutics, have shown promising results in clinical trials, with some scientists calling them "potentially curative." However, concerns remain about the high cost of the treatments, which are expected to cost at least $1 million to $2 million each, and the durability of their benefits.