Chondrial also announced the licensing of a novel investigational technology, CTI-1601, for the treatment of Friedreich’s Ataxia from Indiana University Research and Technology Corp. and Wake Forest Innovations. The CTI-1601 compound was discovered by R. Mark Payne, MD, while an associate professor of Wake Forest School of Medicine, now professor of pediatric cardiology at Indiana University School of Medicine.
Chondrial, which was co-founded by Payne and Steve Plump, will be led by new president and chief executive officer, Carole Ben-Maimon, MD, with the purpose of developing a pipeline of products to treat rare mitochondrial diseases. CTI-1601 is one of the technologies that will be investigated with the Series A investments funds.
CTI-1601 treats Friedreich’s Ataxia, a debilitating, life-shortening, degenerative neuromuscular disease that occurs in about 5,000 people in the United States and about twice that number in Europe. The disease is caused by an abnormal gene inherited in an autosomal recessive fashion and is responsible for the production of frataxin. People with Friedreich’s Ataxia have very low levels of frataxin, which is active in the mitochondria and assists in energy production. These low levels can lead to muscle weakness, loss of coordination, vision impairment and a number of other motor function issues.
CTI-1601 uses a carrier protein to deliver frataxin, the deficient protein in Friedreich’s Ataxia, to the mitochondria where researchers believe it is processed to mature frataxin and becomes active in mitochondrial metabolism. By replacing the deficient protein, mitochondria resume normal function, patients’ symptoms may be minimized and disease progression may be curtailed.
To investigate treatment of Friedreich’s Ataxia, Chondrial acquired technology based on intellectual property owned by Wake Forest Baptist Medical Center and licensed through its commercialization arm, Wake Forest Innovations.
To further support research into CTI-1601, Wake Forest Innovations is also participating in the Series A investment in Chondrial through the Catalyst Fund, which supports activities that can increase the value of a technology as it advances from an idea or invention all the way through its development to licensing.
“These licensing and funding agreements reflect our mission to improve health through collaborative innovation,” says Jeff Brennan, vice president of technology development and commercialization at Wake Forest Innovations.
“We are pleased to be a part of bringing this important discovery closer to positive patient outcomes,” Brennan says. “The Deerfield investment represents precisely the type of ‘multiplier effect’ of additional investments we envisioned when creating the Catalyst Fund.”