ROCKVILLE, Md., Nov. 29, 2023 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX) today announced that the first patient received RGX-202 at dose level 2 in the Phase I/II AFFINITY DUCHENNE® trial. RGX-202 is an investigational one-time AAV Therapeutic for Duchenne muscular dystrophy (Duchenne), using the NAV® AAV8 vector to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain as well as a muscle-specific promoter to support a targeted therapy for improved resistance to muscle damage associated with Duchenne.
“Progressing to dose level 2 is an important milestone in our updated strategic plans and for accelerating the development of RGX-202,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “There is a large unmet need for new therapies for boys with Duchenne, and the market is capable of supporting multiple gene therapies. We believe RGX-202 has unique, differentiating features that support its potential to be a best-in-class product.”








