Chondrial Therapeutics Secures Up to $22.6 Million in Series A Financing and Licenses Novel Technology for the Treatment of Friedreich's Ataxia

PR Newswire, News provided by Chondrial Therapeutics, Inc. Feb 06, 2017, Company Plans to Advance Pipeline of Product Candidates Focused on the Treatment of Rare Mitochondrial Diseases, Carole Ben-Maimon, MD, Named President & CEO

Discovered by Dr. Payne while at Wake Forest Baptist Medical Center, CTI-1601 utilizes 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. People with Friedreich's Ataxia have very low levels of frataxin, which is active in the mitochondria and assists in energy production. 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.

This comes a few months after Horizon Pharma’s Actimmune missed the primary endpoint in a phase 3 trial in the rare neurodegenerative movement disorder, sparking a 20% drop in its stock price.