Rocket Pharmaceuticals received FDA approval for Kresladi, its gene therapy for leukocyte-adhesion deficiency type 1 (LAD1), an ultra-rare inherited immunodeficiency that can be fatal in infants and young children. The approval makes Kresladi the first commercial gene therapy product for this disease indication. The decision adds a new anchor to Rocket’s gene therapy portfolio and demonstrates continued regulatory willingness to advance one-time treatments in small, high-need genetic disorders. For payers and health systems, it also raises the bar for evidence packages and access pathways given the urgency and rarity of the patient population.