FDA Approves Landmark Gene Therapy for Severe LAD-I After Successful UCLA-Led Clinical Trials

The FDA has approved Kresladi, a groundbreaking gene therapy developed at UCLA that restores immune function in children with the rare disorder LAD-I.

By: AXL Media

Published: Mar 28, 2026, 11:08 AM EDT

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

A Historic Victory Against a Rare Genetic Killer

For children born with leukocyte adhesion deficiency-I (LAD-I), the simple act of fighting off a common infection is an impossible biological task. This rare genetic condition, affecting roughly one in a million people, stems from mutations in the ITGB2 gene that prevent white blood cells from reaching the site of an infection. Without the ability to "stick" to blood vessel walls and migrate into tissue, these patients face a childhood defined by recurrent, life-threatening fungal and bacterial outbreaks. However, the FDA’s approval of Kresladi this week marks a turning point in pediatric immunology, providing a definitive, one-time treatment for a disease that previously offered few paths to survival beyond adolescence.

The Mechanics of Stem Cell Rejuvenation

Kresladi functions through an ex vivo gene therapy process that utilizes the patient's own biological material. Doctors harvest blood stem cells from the child and, in a laboratory setting, insert a functional copy of the ITGB2 gene. When these modified cells are returned to the patient, they begin producing white blood cells equipped with the necessary CD11 and CD18 proteins. This localized "factory" allows the body to maintain its own immune defense. Because the therapy uses the patient's own cells rather than a donor’s, it eliminates the devastating risk of graft-versus-host disease, a common and dangerous complication in traditional bone marrow transplants.

Clinical Success and Reduced Toxicity

The FDA approval was anchored by a global clinical trial involving nine patients, six of whom were treated at UCLA. The results were remarkably consistent: every child in the study survived without the need for a bone marrow transplant. Researchers observed a dramatic reduction in hospitalizations for severe infections and a stabilization of white blood cell counts. Dr. Donald Kohn, a pioneer in the field with 30 years of research experience, noted that the therapy is associated with significantly fewer short-term toxicities than traditional treatments, which often require aggressive chemotherapy and long-term immunosuppressive drugs.