Baby Is Healed With World’s First Personalized Gene-Editing Treatment

KJ, a 9½-month-old baby, became the first patient of any age to receive a custom gene-editing infusion designed to fix his unique mutation. Doctors presenting this groundbreaking treatment at a major conference and in a top medical journal suggest its implications extend far beyond KJ alone, offering a new pathway for rare genetic diseases that have long been too costly or inaccessible for potential treatments. This success could revolutionize drug development for conditions like KJ’s, which are caused by single mutations and typically require expensive, lengthy research.