about us

For more than a decade, we’ve forged new paths for gene therapies.

We custom design each of our investigational therapies with the goal of addressing the underlying cause of disease at the genetic level. We are currently developing gene therapies for severe genetic diseases and are advancing research to apply new technologies to these and other diseases. More than 170+ patients have received our therapies across 8 clinical trials.

We seek diversity, yet fly as one

We are 375+ people who take bold risks and collaborate with a broad community to help improve the lives of patients and families. We pride ourselves on a unique, caring, inclusive culture that helps each of us be our best every day. Honesty, respect, and a genuine commitment to access, equity and inclusivity find their way into everything we do.