REGENXBIO Announces Positive Interim Data and Update for PHASE I/II Trial of RGX-121 for the Treatment of MPS II

REGENXBIO  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 2.0x1011 [...]