CTSI collaboration contributed to gene-therapy program


A researcher at the University of Florida has received a $750,000 grant for the development of a gene therapy program aimed at treating Friedreich’s ataxia, a progressive, debilitating and life-shortening neuromuscular disorder, from the Italian patient advocacy group GoFAR, according to this news release.

The researcher, Manuela Corti, P.T., Ph.D., also recently received a clinical grant, $310,000, from the Friedreich’s Ataxia Research Alliance. A CTSI 2014-2015 pilot award and Children’s Miracle Network funds partially funded preliminary data supporting this two-year grant.

“At the University of Florida Clinical and Translational Science Institute, we are very excited to participate in cutting-edge clinical development such as the collaboration with GoFAR,” said David R. Nelson, M.D., assistant vice president for research and director of the CTSI. “Academic and foundation collaboration is the cornerstone of developing innovative therapies in rare disease.”

Researchers will conduct clinical trials in the CTSI’s Clinical Research Center and use the institute’s Quality Assurance services.

Corti, together with UF Powell Gene Therapy Center director Barry Byrne, M.D., Ph.D., will initiate the program.

The GoFAR award to Corti, of the University of Florida Powell Gene Therapy Center, a part of UF Health, will fund an 18-month comprehensive gene therapy program, including both preclinical and planned human studies that address the underlying cause of Friedreich’s ataxia, a genetic disease caused by defects in the frataxin gene. Symptoms of Friedreich’s ataxia usually begin in late childhood and include progressive loss of neuromuscular function and physical coordination. The disorder, which affects about 1 in 50,000 people, can also cause heart disease, diabetes and premature death.

Read the full release here: http://bit.ly/2cuGHN6.