he CAMPSIITE trial reported an 82% median reduction in CSF HS D2S6 sustained through one year. How significant is this level of biomarker reduction in terms of addressing the neurological burden of MPS II?

The study highlighted a strong correlation between week-16 HS D2S6 reduction and neurocognitive outcomes at one year. How convincing is this evidence in validating HS D2S6 as a surrogate endpoint likely to predict long-term clinical benefit?

Pivotal participants showed either stability or continued skill acquisition through one year. In a disease where decline is expected, how clinically meaningful is the preservation or gain of developmental milestones?

Given that existing therapies for MPS II do not address the neurological manifestations, how does RGX-121 compare in terms of its potential to alter the disease course?

No safety-related concerns have been raised across 26 treated patients, and the FDA inspections concluded without observations. How important is this safety record for regulators when considering accelerated approval in such a vulnerable pediatric population?

RGX-121 is designed as a one-time AAV therapy. What are the key challenges and opportunities you see in shifting MPS II treatment from chronic enzyme replacement or supportive care to a one-time genetic approach?

With the BLA under review and an FDA decision expected in February 2026, what are the most important considerations regulators will weigh when evaluating RGX-121 for potential accelerated approval?

If RGX-121 is approved, it would be the first one-time therapy to directly address the genetic cause of Hunter syndrome. How might this outcome influence future gene therapy development for other lysosomal storage disorders?