We Persevere When the Goal is Audacious
Our highest priority is to meet the needs of patients with commitment and compassion.
We strive to be transparent and trustworthy, while taking a strategic and focused approach to our work translating cutting-edge science into new medicines for genetic diseases. We are passionate about what we do and having fun while we do it. We seek to create an inclusive family of science and business professionals striving for success.
Backed by years of expertise
We have assembled an experienced team to develop genomic therapies for rare diseases across all the necessary competencies: regulatory, manufacturing, technical operations, clinical development and commercial strategy.View Leadership