FDA Approves Kresladi Gene Therapy for Rare Immune Disorder

On October 23, 2026, the U.S. Food and Drug Administration (FDA) approved Kresladi, marking a significant advancement in the treatment of severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic immune disorder. This condition affects approximately one in one million children globally and impairs the ability of white blood cells to reach and respond to infections due to mutations in the ITGB2 gene. As a result, affected children face heightened vulnerability to recurrent and often life-threatening infections, making timely treatment essential. The approval of Kresladi is based on promising results from a clinical trial led by Dr. Donald Kohn at UCLA, who has dedicated over 30 years to developing gene therapies for pediatric immune disorders. The trial involved nine patients aged between 5 months and 9 years, reflecting the rarity of LAD-I. All nine trial participants were treated without the need for a bone marrow transplant, and no cases of graft failure or immune rejection were reported. Patients showed a significant reduction in severe infections and improvements in their immune function, indicating the therapy's effectiveness.

Kresladi operates by incorporating a healthy copy of the ITGB2 gene into the blood stem cells of each patient. Once modified, these cells are returned to the child, enabling their bodies to produce functional immune cells capable of combating infections. This innovative approach minimizes the risks associated with traditional donor cell transplants, which often require extensive chemotherapy and immunosuppressive drugs. Dr. Kohn noted that this therapy is associated with fewer short- and long-term toxicities compared to conventional treatments. The FDA approval of Kresladi is a milestone not only for Dr. Kohn but also for the California Institute for Regenerative Medicine (CIRM), which co-funded the clinical trials. This achievement is expected to pave the way for more gene therapies to become commercially available, as Kohn emphasizes the need for greater investment in treatments for rare diseases. He is currently conducting trials for another severe immune disorder, ADA-SCID, which has shown similarly promising results.

Looking ahead, Kresladi is anticipated to be available through specialized treatment centers experienced in gene therapy procedures. The ongoing evaluation of long-term follow-up data from treated patients will further confirm the therapy's clinical benefits. The approval of Kresladi represents a significant step in the development of gene therapies for rare conditions, offering hope to families affected by LAD-I and highlighting the potential for future advancements in the field of regenerative medicine.