Baby Is Healed with First Personalized Gene-Editing Treatment

Instead, KJ has made medical history. The baby, now 9 ½ months old, became the first patient of any age to have a custom gene-editing treatment, according to his doctors. He received an infusion made just for him and designed to fix his precise mutation. The investigators who led the effort to save KJ are presenting their work on Thursday at the annual meeting of the American Society of Cell & Gene Therapy, and are also publishing it in the New England Journal of Medicine. The implications of the treatment go far beyond treating KJ, said Dr. Peter Marks, who was the Food and Drug Administration official overseeing gene-therapy regulation until he recently resigned over disagreements with Robert F. Kennedy Jr., the secretary of health and human services. More than 30 million people in the United States have one of more than 7,000 rare genetic diseases. Most are so rare that no company is willing to spend years developing a gene therapy that so few people would need. But KJ’s treatmen ... Read full article.