Developing disease-modifying precision therapeutics - Positive Functional Modulators (PFMs) - that restore ABC transporter function for patients with serious genetic diseases addressing the underlying cause. ABC transporters are a 48-member family of membrane-bound proteins with etiologic loss-of-function mutations in multiple organ systems and therapeutic areas including the lungs, liver, gastrointestinal tract, eye, and central nervous system. The companyâs pipeline spans multiple therapeutic areas
