We're good humans
who are all-in.

Every day.

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.

Meet our leadership

We’re probably biased, but these are some great minds.

See who we work with

Pursuing gene therapies takes teamwork. Learn about our collaborations.

Learn about how we work

We don’t compromise on our ethics. Read our code of conduct.

Pre teen boy walking down a hallway lined with people while high fiving

Explore our grants & sponsorship programs

We believe in supporting what others are passionate about.

Join the flock

Like what you see? Take a look at our openings.
National Institutes of Health. Genetics Home Reference. Help me understand genetics. Available at: https://medlineplus.gov/download/genetics/understanding/primer.pdf. Accessed April 2022.