Overview

Our commitment to patients is what drives us to do more in our quest to deliver new and potentially curative therapies. We believe gene therapy has the potential to transform lives and our goal is to take it out of the realm of possibility and make it a reality for patients living with devastating diseases.

We are committed to collaborating with patients, physicians, advocacy groups and research centers to gain a deeper understanding of the rare conditions we aim to treat and to improve our ability to customize treatments in order to potentially provide the best possible outcome for patients.

We are grateful for all our partners and participants in our clinical trials. Our work would not be possible without their support.