FDA approves first gene therapy for severe leukocyte adhesion deficiency-I
On October 9, 2026, the FDA approved Kresladi, a one-time gene therapy for severe leukocyte adhesion deficiency-I. Developed at UCLA, the therapy adds a healthy copy of the ITGB2 gene to patients’ blood stem cells, resulting in restored immune function for trial participants. All nine children in the pivotal trial are alive and have experienced significantly fewer severe infections requiring hospitalization.
The U.S. Food and Drug Administration (FDA) has approved Kresladi, marking a significant advancement in the treatment of severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder that affects the immune system. This approval, announced on October 9, 2026, is the culmination of over 30 years of research by Dr. Donald Kohn at UCLA, who has devoted his career to developing gene therapies for pediatric immune disorders. The therapy works by adding a healthy copy of the ITGB2 gene to patients’ blood stem cells, enhancing their immune response and significantly improving their quality of life.
Severe leukocyte adhesion deficiency-I is a rare condition that affects approximately one in one million children worldwide. It is characterized by recurrent infections due to mutations in the ITGB2 gene, which disrupts the function of proteins critical for white blood cell activity. Without effective treatment, children with LAD-I face life-threatening infections that can often lead to early mortality. The approval of Kresladi represents a breakthrough in managing this condition, providing a viable treatment option for affected children.
The pivotal clinical trial involved nine patients aged between 5 months and 9 years, illustrating the rarity of the disorder. Conducted through a collaboration between UCLA and Rocket Pharmaceuticals, which sponsored the research, the trial yielded promising results. Notably, all nine children in the trial survived without requiring bone marrow transplants, showing marked improvements in their immune function and experiencing fewer instances of severe infections that necessitated hospitalization. This positive outcome highlights the therapy's potential to transform the lives of children suffering from this debilitating condition.
Kresladi employs a unique approach by using the patients' own blood stem cells, which reduces the risk of immune rejection associated with donor cells. This innovative strategy not only minimizes the need for extensive chemotherapy but also decreases the long-term side effects typically linked to bone marrow transplants. Additionally, the therapy has demonstrated a significant reduction in leukocytosis, or high white blood cell counts, further indicating its effectiveness in restoring normal immune function.
Looking ahead, the approval of Kresladi is expected to pave the way for further advancements in gene therapies for rare diseases. Dr. Kohn is hopeful that this milestone will encourage additional research and investment in treatments for other rare immune disorders. The therapy is anticipated to become available through specialized treatment centers experienced in ex vivo gene therapy procedures. With ongoing follow-up studies planned to evaluate the long-term effects of Kresladi, this approval signifies a critical step in addressing the unmet medical needs of children with severe LAD-I and similar conditions.
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