Repligen Seeks IND for Drug to Treat Friedreich’s Ataxia

Repligen has submitted an IND for a drug to treat a rare genetic defect that causes the body to produce too little of a protein central to healthy nerve function. If approved, RG2833, an oral selective histone deacetylase 3 inhibitor, would become the first drug to treat Friedreich’s ataxia. The inherited neurodegenerative disease is caused by a defect in a single gene that results in inadequate production of the protein frataxin.
Washington Drug Letter