Gene-editing treatments are currently being developed for several rare and inherited genetic diseases. One gene-editing treatment, called Casgevy, is approved and available commercially to treat sickle cell disease and a related blood disorder called beta thalassemia. Earlier this year, KJ Muldoon, a baby boy born with an often fatal genetic disease that causes ammonia to build up in his blood, was saved with a customized gene-editing treatment—a medical first. Global Genes, a rare disease advocacy organization, estimates that there are at least 10,000 rare diseases that affect more than 400 million people worldwide. “We urgently need these ways to directly treat the root cause of these genetic diseases.”