Rocket Pharma Wins FDA Approval For KRESLADI — The First Ever Gene Therapy For Severe LAD-I
Published on March 27, 2026.
Rocket Pharmaceuticals Inc. has received accelerated approval for KRESLADI, the first ever gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I). The approval is based on clinical data showing the restoration of CD18 and CD11a expression on neutrophils, improved immune function, and clinical benefits in treated pediatric patients. KRESlADI received multiple expedited designations including Orphan Drug, Rare Pediatric Disease, Regenerative Medicine Advanced Therapy (RAT), and Fast Track. As part of the accelerated approval, Rocket must conduct confirmatory studies to verify long-term clinical benefit.
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