RG2833: Potential to Alter the Course of Friedreich's Ataxia
Repligen is currently developing RG2833, an inhibitor of histone deacetylase (HDAC Class 1), for the treatment of inherited neurodegenerative diseases such as Friedreich's ataxia. Friedreich's ataxia is caused by inadequate production of the protein frataxin and leads to degeneration of the nerves controlling muscle movements in the arms and legs and the nerve tissue in the spinal cord.
Preclinical Development Program
Preclinical studies have shown that HDAC (Class 1) inhibitors increase production of the protein frataxin which may have the potential to arrest disease progression in patients with Friedreich's ataxia. Pending regulatory approval, Repligen plans to initiate a Phase 1 study in patients in Europe later this year. We have developed a biomarker to measure changes in frataxin levels in patient cells for use in our clinical trials which may enable us to gain early insight into the potential benefit of teating patients with RG2833.
Our research efforts have been funded in part with grants from the Muscular Dystrophy Association (MDA), the Friedreich's Ataxia Research Alliance (FARA), Go FAR (Friedreich's Ataxia Research) and the National Ataxia Foundation (NAF).
About Friedreich's Ataxia
Friedreich's ataxia is an inherited neurodegenerative disease caused by a single gene defect that results in inadequate production of the protein frataxin. Low levels of frataxin lead to degeneration of both the nerves controlling muscle movements in the arms and legs and the nerve tissue in the spinal cord. Symptoms of Friedreich's ataxia typically emerge between the ages of five and 15 and often progress to severe disability, incapacitation or loss of life in early adulthood. There are approximately 15,000 patients worldwide with Friedreich's ataxia.