A Find-and-Replace Approach to Fixing the Mutation Underlying Sickle Cell Disease

Gene editing is an emerging therapeutic area that promises to correct the underlying genetic causes of diseases. Graphite Bio is readying to enroll its first patient in a phase 1/2 clinical study of its experimental gene editor GPH101 to correct the mutation in the beta-globin gene that drives sickle cell disease. Though the condition can manifest itself differently from patient to patient, it can cause painful episodes due to the clumping of sickle-shaped blood cells that obstruct blood flow in small blood vessels, as well as other acute complications including stroke and infections that can contribute to early mortality in these patients. We spoke to Josh Lehrer, CEO of Graphite Bio, about the company’s experimental sickle cell gene editing therapy, how it works, and what makes it a next-generation gene editor.