ROCKVILLE, Md., Dec. 8, 2020 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced interim data from 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). In addition, REGENXBIO announced plans to evaluate a higher dose of RGX-121 in a third cohort of patients at an increased dose of 2.0×1011 GC/g brain mass. REGENXBIO expects to begin enrolling patients in Cohort 3 in the first quarter of 2021.
{iframe}https://www.biospace.com/article/releases/regenxbio-announces-positive-interim-data-and-update-for-phase-i-ii-trial-of-rgx-121-for-the-treatment-of-mps-ii/{/iframe}