Our Pipeline


 Our Rare Disease Pipeline

  • Duchenne Muscular Dystrophy

    Phase 3 ongoing
  • Non-Ambulatory DMD

    Designing Phase 2
  • Becker Muscular Dystrophy

    Exploring potential in BMD
  • Cystic Fibrosis

    CAT-5571 is an oral small molecule designed to restore host defense by activating autophagy being developed for the treatment of cystic fibrosis (CF). Autophagy is a mechanism for recycling cellular components and digesting pathogens, which is depressed in CF. People with CF suffer from persistent lung infections with opportunistic pathogens such as P. aeruginosa and B. cenocepacia, causing chronic infections that are difficult to eradicate and ultimately lead to respiratory failure. CAT-5571 has been shown to restore autophagy, reestablish host defense and enhance the clearance of pathogens, including P. aeruginosa and B. cenocepacia, in preclinical models of CF.