The FDA approved Rocket Pharma’s gene therapy Kresladi for severe leukocyte adhesion deficiency type 1 (LAD-1), a highly ultra-rare disorder that exposes children to life-threatening infections. The therapy, which had faced an FDA rejection in 2024 over manufacturing concerns, is now cleared for children without a matched sibling stem-cell donor. Rocket said it expects only a limited number of U.S. patients annually, which is reflected in the approval’s narrower commercial scale despite the potential for major clinical benefit. Kresladi’s approval also highlights how manufacturing concerns can become decisive during gene therapy regulatory reviews, particularly for late-stage manufacturing readiness.