FDA Grants Landmark Approval to Kresladi as First Gene Therapy for Rare Pediatric Immune Disorder LAD-I

The FDA has approved Kresladi, the first gene therapy for pediatric LAD-I, using a patient’s own stem cells to restore immune function and prevent infections.

By: AXL Media

Published: Mar 28, 2026, 5:08 AM EDT

Source: Information for this report was sourced from University of California - Los Angeles Health Sciences

A Scientific Milestone for Pediatric Immunology

The FDA’s authorization of Kresladi represents a historic shift in the treatment of Leukocyte Adhesion Deficiency-I (LAD-I), a condition that has long been a death sentence for affected infants. This genetic disorder, caused by mutations in the ITGB2 gene, prevents white blood cells from adhering to and exiting blood vessels to combat infections. By successfully navigating the rigorous federal approval process, this therapy moves from the realm of experimental clinical trials into a standardized medical reality, providing a definitive therapeutic pathway for a disease that previously offered few survivors beyond childhood.

The Mechanism of Autologous Stem Cell Modification

At the core of the Kresladi platform is an ex vivo gene therapy process that utilizes the patient’s own blood stem cells. Doctors extract these cells and insert a functional copy of the ITGB2 gene before reinfusing them into the child’s body. This autologous approach inherently eliminates the risk of graft-versus-host disease, a common and often fatal complication associated with traditional donor bone marrow transplants. According to Dr. Donald Kohn of UCLA, using the patient's own cellular material significantly reduces the need for the aggressive chemotherapy and long-term immunosuppression typically required when introducing foreign donor cells.

Clinical Success and Patient Survival Rates

The data supporting the FDA’s decision was derived from a global clinical trial where every participant survived without the need for a follow-up bone marrow transplant. The trial cohort, while small due to the extreme rarity of LAD-I, demonstrated a marked reduction in hospitalizations and life-threatening bacterial or fungal infections. Researchers observed a sustained expression of CD18 and CD11a proteins, which are the critical components necessary for white blood cell mobility. These biological markers remained stable over long-term follow-up, suggesting that the one-time intervention provides a durable, potentially lifelong correction of the underlying immune deficiency.