(RTTNews.com) - Repligen Corp. (RGEN) said Thursday it has enrolled its first patient in a Phase 1 clinical trial of RG2833 in adult patients with Friedreich's ataxia, or FA.
FA is an inherited neurodegenerative disease caused by low levels of the protein frataxin which results in symptoms that typically present in childhood and lead to progressive loss of muscle and nerve function, often resulting in loss of life by early adulthood.
RG2833 is an orally bioavailable, class 1 histone deacetylase inhibitor (HDACi) specifically 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.
The Phase 1 trial is a single ascending dose, crossover study in up to 20 adult FA patients. It is designed to evaluate the pharmacokinetic and safety profile of RG2833.
Repligen has previously received U.S. Orphan Drug and European Orphan Medicinal Product designations for RG2833 for the treatment of FA.
For comments and feedback: contact editorial@rttnews.com
Originally posted here:
Repligen Enrolls First Patient In Study Of RG2833 To Treat Friedreich's Ataxia