Who We Are

Pioneers in Gene Therapy

Since its founding in 2009 by Ken Mills and FOXKISER, 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.

Today, we have end-to-end capabilities in the development of AAV Therapeutics and are advancing our internal pipeline to treat common and rare diseases. 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.

We know that every day matters to patients and their families, and that’s why we work with urgency.