Waltham, Mass.-based Repligen has enrolled the first patient in a phase I clinical trial of RG2833 in adult patients with Friedreich’s Ataxia (FA)—a debilitating, life-shortening, degenerative neuro-muscular disorder that currently has no treatment.

Repligen’s RG2833 is a class 1 Histone Deacetylase (HDAC) inhibitor specifically designed to increase frataxin protein production in FA patients, targeting the genetic mutation that is the fundamental cause of the disease. The potential for HDAC inhibitors as a therapy for FA was first demonstrated by Dr. Joel Gottesfeld and his colleagues at Scripps Research Institute in La Jolla, Calif., whom Repligen has worked with to optimize and develop their own HDAC inhibitors and prepare them for human clinical trials.

"Friedreich's Ataxia disease biology provides evidence that a small increase in expression of the defective gene could potentially slow disease progression," said James R. Rusche, Ph.D., senior vice president, R&D, Repligen. "RG2833 is the first compound that targets activation of this defective gene. If our unique approach of using small molecules for protein replacement is successful, it has the potential to significantly improve outcomes for patients with FA.”

Rusche added, “We are hopeful that the phase I trial will elucidate the role for HDAC inhibition in FA, and inform future efficacy studies."