REGENXBIO(US:RGNX) Prnewswire·2025-03-10 11:05Group 1 - REGENXBIO Inc. announced new interim biomarker data from the Phase I/II portion of the AFFINITY DUCHENNE® trial for RGX-202, aimed at treating Duchenne muscular dystrophy, to be presented at the 2025 MDA Clinical & Scientific Conference [1] - The presentation will include interim clinical data on RGX-202 and a discussion on the enhanced therapeutic potential of a microdystrophin with an extended C-terminal domain [2] - REGENXBIO is a leading clinical-stage biotechnology company focused on gene therapy, with a pipeline that includes treatments for rare and retinal diseases [3] Group 2 - The company has developed AAV Therapeutics, an innovative class of gene therapy medicines, and has treated thousands of patients with its platform [3] - Notable products in development include RGX-202 for Duchenne muscular dystrophy, ABBV-RGX-314 for wet AMD and diabetic retinopathy in collaboration with AbbVie, and RGX-121 for MPS II [3] - AAV Therapeutics are designed as one-time treatments, potentially transforming healthcare delivery for millions [3]