REGENXBIO Announces Positive Data from Pivotal Dose Level of RGX-121 Demonstrating Long-Term Systemic Effect
REGENXBIO(RGNX) Prnewswire·2024-09-03 12:04- Data from pivotal dose level demonstrates long-term, sustained reductions in CSF levels of HS D2S6, a key biomarker of brain disease in MPS II - 80% of patients who received the pivotal dose discontinued intravenous enzyme replacement therapy or remained treatment-naïve - Submission of a rolling BLA using the accelerated approval pathway on track for Q3 2024 ROCKVILLE, Md., Sept. 3, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced positive results from the Phase I/II/III CAMPSIITE® trial ...