Our Research

In developing much-needed therapies for patients with serious rare and ultra-rare genetic diseases, we match the best treatment modality to each disease.

Our diverse portfolio of approved medicines and investigational therapies is advancing first- or best-in-class treatments for diseases that exhibit a clear biology for treatment and are severely underserved by current standard of care.

Our proprietary manufacturing platform can produce AAV vectors with extraordinary efficiency and lower material costs than traditional methods, and at a scale to meet today’s demands.