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ROCKVILLE, Md., Feb. 8, 2021 /PRNewswire/ REGENXBIO Inc. (Nasdaq: RGNX) today announced that additional positive interim data from a total of eight patients in Cohorts 1 and 2 of the ongoing Phase I/II trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome, will be presented at the 17
th Annual WORLD
Symposium™. We are pleased to report additional positive data from Cohorts 1 and 2 of our ongoing Phase I/II trial of RGX-121. Data from the eight patients dosed to date continue to show an encouraging safety profile of RGX-121, and highlight the potential of RGX-121 to restore biological enzyme activity and improve outcomes for MPS II patients, said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. We look forward to enrolling patients in Cohort 3 at a higher dose level of RGX-121, which we expect to begin in the first quarter of 2021. �