REGENXBIO Inc.

Historically, the primary challenge for gene therapy has been the safe and effective delivery of genes into cells. To address this challenge, scientists designed and developed a variety of gene vectors to facilitate gene delivery into cells.

Our gene therapies are designed to deliver working genes to cells using AAV vectors as the delivery “vehicles.” We chose AAV vectors, modified viruses that cannot increase their numbers or reproduce themselves, based on several factors including their demonstrated efficiency in gene delivery to date and promising safety profiles for gene therapy.

Since our inception, we have built and advanced multiple promising gene therapies into late-stage clinical development. All of our gene therapies were built on our NAV Technology Platform. Together, with our in-house, U.S.-based manufacturing and end-to-end capabilities, we are excited about the prospect of bringing these assets to commercial stage and continuing to leverage our NAV Technology Platform to generate additional promising gene therapies.