Mitochondrial disorders are common and are very difficult to treat. Our mission is to develop therapies for these diseases. We are initially targeting Friedreich’s Ataxia (FA), which is a progressive disease that affects multiple body systems, particularly the brain and heart. FA is the most common ataxia in man and is caused by a deficiency of a key protein, frataxin, which is normally present in the mitochondria of all tissues. Children and young adults progressively lose the ability to walk and speak, and their hearts become very thick and weak, and can eventually fail. There is currently no cure for FA.
Discovered by Dr. Payne while at Wake Forest Baptist Medical Center, CTI-1601 utilizes a carrier protein to deliver frataxin, the protein deficient in Friedreich’s Ataxia, to the mitochondria where researchers believe it is processed to mature frataxin and becomes active in mitochondrial metabolism.
Chondrial’s premise in the research program is that by replacing the deficient protein the mitochondria will resume normal function, patients’ symptoms may be minimized, and disease progression may be curtailed.
CTI-1601 is currently being evaluated in a Phase 1 clinical trial for the potential treatment of patients with Friedreich’s ataxia.
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