Repligen Corp. (NASDAQ:RGEN) in Waltham has enrolled (March 2012) its first patient in a Phase 1 clinical trial of RG2833 in adult patients with Friedreich’s ataxia (FA), see
Repligen also reported it’s year-end financial results for 2011, according to two announcements this week.
The RG2833 drug candidate is an oral class 1 histone deacetylase inhibitor (HDACi) designed to increase frataxin production in patients with FA. The study is being conducted in Turin, Italy and is the first clinical trial of a drug that targets the core genetic defect in FA.
“RG2833 is an attractive drug candidate, given its oral bioavailability and potential to target and activate the defective gene responsible for Friedreich’s ataxia,” said lead investigator Prof Luca Durelli, M.D., chief of neurology at San Luigi Gonzaga University Hospital in Turin, Italy and lead investigator for the Phase 1 trial. “The effects of FA are devastating for our young patients and their families.”