Since our founding in 2009, REGENXBIO has been a leader in the gene therapy field, driven by our ongoing commitment to develop gene therapies that improve treatment options for people with significant unmet needs.
In our early days, we focused on licensing our NAV® Technology Platform to other biotechnology companies. This included licensing NAV® vectors to leading pharma companies and supporting the formation of new companies based on our technology. In 2019, the first gene therapy utilizing NAV® Technology was approved by the US FDA for the treatment of Spinal Muscular Atrophy. It was one of only two gene therapies approved at the time.
Today, we are advancing a late-stage clinical pipeline of gene therapies for rare and retinal diseases. These programs are designed to maximize the potential of therapeutic benefit for patients. We do this through innovative constructs, strategic delivery methods and a focus on patient safety.
At our FDA-inspected Manufacturing Innovation Center in our Rockville, MD headquarters, we are optimizing purity, productivity, and manufacturability of our investigational gene therapies at commercial scale.
We know that every day matters to patients and their families. That’s why we work with urgency towards our goal of discovering and delivering new, meaningful medicines.