RGX-202 is an investigational, one-time AAV gene therapy designed to deliver an optimized microdystrophin gene encoding for a novel microdystrophin protein that includes the C-Terminal (CT) domain found in naturally occurring dystrophin. AFFINITY DUCHENNE®, a phase I/II/III multi-center, open-label trial of a one-time intravenous administration of RGX-202 is enrolling ambulatory boys aged one and above with Duchenne to evaluate safety, tolerability, and clinical efficacy. This program will share details of the trial for potential referral of patients to AFFINTY DUCHENNE trial sites.

Speakers:

• Stephen Pakola, MD, Chief Medical Officer; moderator; spakola@regenxbio.com
• Jahannaz Dastgir, DO, REGENXBIO, Sr Medical Director, Clinical Development, DMD; jdastigr@regenxbio.com
• Nancy Kuntz, MD, Lurie Children’s Hospital, Professor Pediatrics & Neurology, AFFINITY DUCHENNE Primary Investigator, nkuntz@luriechildrens.com