Latest news

REGENXBIO to receive $110 million upfront, potential milestone payments of up to $700 million and meaningful double-digit royalties on net sales, and lead manufacturing Nippon Shinyaku to lead commercialization of first potential gene therapies for Mucopolysaccharidosis II (MPS II) and

Pivotal data evaluating the safety and efficacy of the subretinal delivery of ABBV-RGX-314 in patients with   wet age-related macular degeneration (wet AMD) are expected in 2026 AbbVie and REGENXBIO will plan the Phase 3 clinical program of investigational ABBV-RGX-314 in diabetic retinopathy (DR)

Alignment achieved with FDA on AFFINITY DUCHENNE ® pivotal program and access to accelerated approval; BLA expected in 2026 Pivotal trial of RGX-202 is enrolling ambulatory patients aged 1 and above with first patient dosed Phase I/II data show RGX-202 recipients exceeding external natural history

Arshad M. Khanani, MD, MA, FASRS

Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil

Jahannaz Dastgir, DO, Sr Clinical Development Lead