FDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder

MedicalXpress | 三月 28, 2026

Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug Administration–approved therapy for severe leukocyte adhesion deficiency-I—a genetic condition characterized by recurrent infections and, often, early death.