Rocket Pharmaceuticals received FDA approval for Kresladi, positioning the therapy as the first commercial gene therapy for leukocyte-adhesion deficiency type 1, an ultra-rare inherited immunodeficiency that can become fatal in infancy. The approval adds another gene therapy product to the market and extends Rocket’s commercial footprint following its earlier clinical development milestones. The company’s approval signals regulatory confidence in the therapy’s benefit-risk profile for a disorder with limited treatment options. For gene therapy developers, approvals for ultra-rare indications also influence payer coverage decisions and broader adoption pathways. Rocket’s approval also matters for the competitive landscape, where manufacturers are increasingly tied to manufacturing scalability and post-approval outcomes tracking as the category grows.