REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of RGX-202, a differentiated investigational gene therapy for Duchenne muscular dystrophy. The company said the positive biomarker data in patient aged 1-3 add to consistent, robust microdystrophin and transduction levels across all treated ages.
"The new data from the age 1-3 cohort builds on the favorable safety and efficacy profile seen in ages 4 and older and reinforces the potential of RGX-202 to serve a wide age range of patients," said Steve Pakola, Chief Medical Officer of REGENXBIO.
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