Regenxbio presented promising one-year follow-up data for its gene therapy targeting Hunter syndrome, a rare lysosomal storage disorder characterized by iduronate-2-sulfatase deficiency. The data, encompassing 13 patients, demonstrated sustained therapeutic benefit, heightening expectations for eventual FDA approval following a review extension. The therapy employs the company’s NAV Technology platform for targeted delivery, aiming to provide a transformative, one-time treatment alternative to enzyme replacement therapy. Pending regulatory feedback, this advancement could finalize pivotal approval for a long-awaited rare disease treatment, aligning with the agency’s increasing focus on innovative genetic therapies.