Chondrial Therapeutics

Chondrial Therapeutics is a clinical-stage biotechnology company developing CTI-1601, a recombinant fusion protein designed to deliver human frataxin to mitochondria in patients with Friedreich’s ataxia (FA), addressing the root cause of the disease. The Phase 1 trial is evaluating safety, tolerability, and pharmacokinetics of subcutaneously administered CTI-1601 in adult FA patients. Positive preclinical data supported trial initiation; topline data were expected by end of 2020. The U.S. FDA has granted CTI-1601 Rare Pediatric Disease and Fast Track Designations, and the program is the only frataxin replacement therapy in clinical development for FA.