Seeking Gene Therapy Cures
Advancing an integrated pipeline of genetic therapies that correct the root cause of complex and rare life-threatening childhood disorders
Mission, Vision & Values
Our mission is to develop first-in-class and best-in-class, curative gene therapies for patients with devastating diseases. Our vision is “Seeking Gene Therapy Cures”. Our company values of Trust, Be Generous, Be Curious and Elevate are at the heart of all that we do.
Our team strives to put patients and their families first when developing treatments for rare and complex inherited childhood disorders. We aim to create a community where rare disease patients and their families can come together and feel supported through treatment and beyond.