The FDA granted accelerated approval to Rocket Pharmaceuticals’ gene therapy Kresladi for severe leukocyte adhesion deficiency type 1 (LAD-1), clearing a pathway after an earlier rejection tied to manufacturing concerns. The therapy is intended for children who lack a matched sibling for stem cell transplant. Rocket’s approval follows a 2024 FDA refusal centered on manufacturing issues, underscoring how technical compliance can become the gating factor for late-stage gene therapy programs. While Rocket has positioned the drug for clinical benefit in an ultra-rare population (estimated around 25 new cases per year), investors have generally viewed Kresladi as more strategic than a near-term revenue engine. Clinically, the decision matters because it validates Rocket’s ability to remediate production constraints and re-enter regulatory review in a disease area where there are limited therapeutic options.