By harnessing our deep experience with well-defined drug development and regulatory pathways, we will rapidly advance our lead programs for cystic fibrosis (CF) caused by nonsense mutations and primary ciliary dyskinesia (PCD) – ultimately having a significant effect where there is a clear, unmet, and high-hurdle need.
CF and PCD are complex diseases to treat, as targeted delivery and restoration of function are big hurdles to overcome. Considering these patients are coping with a compromised quality of life and facing a significantly shortened lifespan, we must meet this need.
Our programs in CF and PCD are currently in preclinical stages of development. Our CF program is supported by a grant from the Cystic Fibrosis Foundation, whose support has allowed us to better understand the pathogenesis of CF and develop a novel therapeutic approach for correcting nonsense mutations for this severe disease.