Posted by Michael Wonder on 18 Dec 2019
Chondrial Therapeutics announces dosing of first patients in Phase 1 clinical program of CTI-1601 for treatment of Friedreich’s ataxia; CTI-1601 granted rare pediatric disease designation and fast track designation by U.S. FDA
18 December 2019 - Chondrial Therapeutics, a clinical-stage biotechnology company focused on developing treatments for rare diseases, with an initial focus on Friedreich’s ataxia, today announced that the first patients have been dosed in a Phase 1 clinical trial to evaluate the safety and tolerability of single ascending doses of CTI-1601.
CTI-1601 is a recombinant fusion protein intended to deliver human frataxin to patients with Friedreich's ataxia, who have decreased levels of frataxin.
Chondrial also announced that the U.S. FDA has granted CTI-1601 rare paediatric disease designation and fast track designation.
