Yesterday, REGENXBIO shared important updates on their Phase I/II AFFINITY DUCHENNE® trial of RGX-202 for Duchenne during their third quarter financial results update. RGX-202 utilizes a novel adeno-associated virus (AAV8) to transport a shortened version of the dystrophin gene (micro-dystrophin) that may provide benefit in place of the missing full-length dystrophin protein.

Key highlights of the company update include the dosing of the last patient in their AFFINITY DUCHENNE trial pivotal dose cohort (ages 4–11) and the first patient in the younger cohort (ages 1–3). The company plans on sharing information on their pivotal trial design and accelerated approval plans this month, in addition to functional data from both dose levels of the AFFINITY DUCHENNE trial.

To learn more about the full program update, join PPMD and REGENXBIO for a community webinar on Wednesday, November 20th at 1:00 PM ET. Register here.