Our proprietary non-viral gene therapy platform

We believe that the properties of our therapies may allow us to initiate treatment in childhood, enabling a greater therapeutic benefit throughout the patient’s life. Our therapies may also be used to treat patients who are not able to receive current gene therapy treatments due to pre-existing immunity, as well as patients who did not see desired efficacy after being treated with other current gene therapies.

Broad patient reach

Groundbreaking science. Trailblazing team.

The people behind our revolutionary gene therapies