Denali Therapeutics received FDA approval for Avlayah, a therapy for Hunter syndrome that leverages Denali’s proprietary delivery technology designed to cross the blood-brain barrier. The approval adds to a growing set of FDA decisions for therapies that can reach the central nervous system in rare neurological conditions. Denali’s platform approach is intended to enable systemic administration of biologics while improving access to brain targets—an area that has historically limited treatment options for MPS disorders. The decision arrives after a period of scrutiny and tightening expectations for rare-disease programs, making regulatory success a key signal for the platform’s translational credibility. For developers, the approval reinforces the commercial and scientific value of delivery systems that reliably reach hard-to-access tissues, particularly in pediatric neurodegenerative and neuroinflammatory diseases where outcomes can hinge on CNS exposure.