REGENXBIO (RGNX) announced that AFFINITY DUCHENNE, the multi-center, open-label trial of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy, has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety data from the Phase I/II portion of the study, including the first functional data. Affinity Duchenne data updates: Results include 12-month data from three dose level 1 patients aged 4-10 and nine-month data from two dose level 2 patients aged 8 and 12. RGX-202 was well tolerated with no serious adverse events and no AEs of special interest. Phase I/II data show RGX-202 recipients exceeding external natural history and established benchmarks for clinical outcomes: Functional improvements seen in all patients treated with dose level 1 and dose level 2 at 12 and 9 months respectively; New biomarker data confirms consistent robust expression of differentiated RGX-202 microdystrophin in the muscle; Favorable safety profile observed at both dose levels; no serious adverse events or AEs of special interest
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