The more people we reach, the more our impact can grow.
In 1997, we were founded to make a big difference in small patient populations. Now we seek to make an even greater impact by applying the same science-driven, patient-forward approach that propelled our last 25 years to larger genetic disorders, as well as genetic subsets of more common conditions. The end goal has always been better lives and now we can reach more.
Patients inspire and enable our work every day. Meet some of the exceptional people whose stories remain at the heart of everything we do.Learn More about our patients ⌃
Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.Learn More about our pipline ⌃