Clinical Trial SuccessPositive 12-month data from the Phase 1/2/3 CAMPSIITE trial support the use of CSF HS D2S6 as a surrogate endpoint, indicating likely clinical benefit under the accelerated approval pathway.
Commercial PotentialIf approved, RGX-121 would be the first and only potential one-time, commercially-available therapy designed to directly address the underlying genetic cause of Hunter syndrome.
Regulatory AdvancementsRGX-121 has received Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, enhancing its regulatory profile.