REGENXBIO Inc.

We are a leading clinical-stage biotechnology company focused on improving lives through gene therapy. Our investigational gene therapies are designed to deliver functional genes to address genetic defects in cells, enabling the production of therapeutic proteins or antibodies intended to impact disease. These therapies use adeno-associated virus (AAV) vectors from our proprietary NAV® Technology Platform, which includes exclusive rights to a large portfolio of AAV vectors (NAV Vectors), such as AAV8 and AAV9. AAV vectors are non-replicating viral delivery vehicles not known to cause disease. Our NAV Technology Platform has been the foundation for commercial and investigational AAV therapeutics that have treated thousands of patients through our clinical pipeline and NAV licensees. We are currently focusing our internal development pipeline on retinal, neuromuscular, and neurodegenerative diseases. Our investigational gene therapies include Surabgene lomparvovec (sura-vec, ABBV-RGX-314), which we are developing in collaboration with AbbVie to treat wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR). We are also developing RGX-202 to treat Duchenne muscular dystrophy (Duchenne).