Change the future
of medicine, of lives

Change the future
of medicine, of lives

Our vision is to enable every patient with a genetic disease to have access to life-changing treatments in their lifetime.

To do so, we must enter a new era of precision medicine - one in which safer, more effective medicines can be optimized to treat a broad range of genetic conditions.

Our team shares a commitment to deliver on the promise of precision medicine for the many patients and families battling devastating diseases.

We are:

Research-driven

Focused on how we can unlock science to cure disease, both with our own labs and through unique partnerships that can expand the impact of our platform.

A learning culture

As a science-driven organization, we believe that freedom to fail is one of the best stepping stones to knowledge. We welcome those who are simultaneously unafraid to challenge convention and humble enough to always keep learning.

Diverse

Our team is both thoughtful and dynamic, bringing deep expertise and unique perspectives to their work.

Purposeful

We work thoughtfully and with a sense of responsibility to advance medicines that will have a meaningful impact on patients' and caregivers' lives.

Corporate Sustainability Matters

We understand that delivering on our vision over the long-term requires a focus on corporate sustainability, including environmental, social and governance ("ESG") considerations.

Read our Proxy statement

We understand that delivering on our vision over the long-term requires a focus on corporate sustainability, including environmental, social and governance ("ESG") considerations.

Read our Proxy statement