Rectifying function, restoring hope. Developing disease-modifying precision therapeutics for patients with serious genetic diseases.
Rectify is developing Positive Functional Modulators (PFMs), disease-modifying precision therapeutics that restore ABC transporter function for patients with serious genetic diseases. 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.
