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 2.0×1011 GC/g brain mass. REGENXBIO expects to begin enrolling patients in Cohort 3 in the first quarter of 2021.
Key highlights from this press release include:
– Consistent reductions in CSF levels of heparan sulfate up to 2 years after administration, as well as continued developmental skill acquisition
– Initial data from Cohort 2 indicates evidence of systemic enzyme expression and biomarker activity
– Additional data from Cohorts 1 and 2 to be presented at WORLD Symposium in February 2021
– REGENXBIO expects to initiate dosing of patients in Cohort 3 at an increased dose in the first quarter of 2021
Read the entire press release here