Dr. Mark Payne, who first described the possibility of frataxin replacement therapy via TAT-Frataxin, has been developing this exciting prospect for F A therapy. Synthetic frataxin protein is partnered with a unique delivery system (a protein fragment called a Trans-Activator of Transcription or TAT) to get the frataxin protein to the mitochondria. Dr. Payne has tested TAT-frataxin in the F A mice and demonstrated proof of principle and compelling results. (See publication below.)
Dr. Payne’s approach increases the life span and weight of F A mice and improves their cardiac function.
Dr. Payne formed a company, Chondrial Therapeutics, to further develop this approach and generate preclinical data. Chondrial is now Larimar Therapeutics and clinical trials of CTI-1601 are in progress.