The year has become a watershed moment for gene editing medicine, with CRISPR treatments now curing diseases that were once considered lifelong sentences and providing hope for millions of patients worldwide. Casgevy, the first approved CRISPR therapy, has already transformed lives for patients with sickle cell disease and beta thalassemia across 50 treatment sites in North America, Europe, and the Middle East, with results showing complete freedom from painful crises that once sent patients to the hospital regularly. Even more remarkable, doctors recently delivered the first-ever personalized CRISPR treatment to an infant in just six months, creating a bespoke therapy tailored specifically to one child’s unique genetic condition.
The success stories are mounting quickly, with new treatments showing 86% reductions in symptoms for rare hereditary diseases and some patients remaining attack-free for over 13 months after just a single treatment. What makes these breakthroughs so revolutionary is that many represent functional cures rather than lifelong management, offering patients the chance to live normal lives without constant medication or medical intervention. The speed of progress has surprised even the researchers, with multiple new CRISPR therapies advancing to late-stage trials and showing dramatic, durable results that are rewriting the textbooks on what’s possible in medicine. This technology represents hope for the 7,000 known rare diseases, most of which have never had effective treatments until now.