FDA approves gene therapy for severe leukocyte adhesion deficiency‑I

The FDA granted accelerated approval to Kresladi, a one‑time gene therapy for severe leukocyte adhesion deficiency‑I, based on phase 1‑2 trial results showing sustained immune reconstitution in treated children. The trial was led by Dr. Donald Kohn at UCLA and sponsored by Rocket Pharmaceuticals.

FDA approval of Kresladi was announced under the accelerated approval pathway for severe leukocyte adhesion deficiency‑I, a rare pediatric immune disorder. The condition is caused by mutations in the ITGB2 gene that disrupt CD11 and CD18 proteins and impair white blood cell responses to infection. The approval relied on outcomes from a phase 1‑2 trial led by Dr. Donald Kohn at UCLA and sponsored by Rocket Pharmaceuticals. Trial reports described sustained immune reconstitution and reductions in severe infections among treated children. Key details: - The clinical trial enrolled nine patients aged 5 months to 9 years, with six treated at UCLA and three treated in London and Spain. - All nine trial patients survived without needing a bone marrow transplant, and no graft failure or immune rejection was reported. - The one‑time, ex vivo therapy adds a healthy copy of the ITGB2 gene to each patient’s blood stem cells, restoring CD11/CD18 expression and improving immune function. - The California Institute for Regenerative Medicine co‑funded the clinical trials, and Rocket Pharmaceuticals sponsored the study. - Kresladi is expected to be offered through specialized centers experienced in ex vivo gene therapy procedures, and confirmation of clinical benefit will depend on longer‑term follow‑up data and a post‑marketing registry. Summary: The FDA approval makes Kresladi the first U.S. gene therapy approved for severe leukocyte adhesion deficiency‑I and follows trial findings of restored immune function and fewer severe infections in treated children. The therapy’s wider availability is expected through specialized treatment centers, and ongoing longer‑term follow‑up and post‑marketing data will be used to confirm clinical benefit.