Regenxbio presents additional positive interim data from phase i/ii trial of rgx-121 for the treatment of mps ii (hunter syndrome) at 18th annual worldsymposium™ 2022

Rockville, md., feb. 9, 2022 /prnewswire/ -- rgx-121, a one-time gene therapy for mps ii, continues to be well-tolerated with no drug-related saes across three dose levels dose-dependent reductions in csf biomarkers observed; cohort 3 patients approach normal levels of d2s6 biomarker  measures of neurodevelopmental function from patients in cohorts 1 and 2 demonstrate continued developmental skill acquisition up to two years after rgx-121 administration  evidence of systemic enzyme expression and biomarker activity continues to be observed  cohort 3 expansion using commercial-scale cgmp material is planned to start in 1h 2022 regenxbio inc. (nasdaq: rgnx) today announced additional positive interim data from cohorts 1-3 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, are being presented at the 18th annual worldsymposium™.
RGNX Ratings Summary
RGNX Quant Ranking