The FDA cleared Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel) as the first gene therapy for severe leukocyte adhesion deficiency type I (LAD-I), granting accelerated approval for pediatric patients without a matched sibling donor for stem cell transplant. The approval comes on the heels of a prior FDA rejection for manufacturing issues. In a separate diabetes milestone, the FDA approved Novo Nordisk’s once-weekly basal insulin Awiqli (insulin icodec) for type 2 diabetes. The approval marks a first-in-class option in the U.S. for weekly dosing and establishes a new competitive baseline for long-acting insulin administration. Together, the two decisions underscore how regulators are continuing to expand therapeutic modalities—both transformative gene therapy and new dosing schedules—while setting expectations for manufacturing reliability and clinical durability.