Repligen Reports Identification Of Drug Target For Friedreich’s Ataxia

Repligen has announced the publication of research that identifies histone deacetylase 3 (HDAC 3) as an enzyme target for therapeutic intervention in Friedreich’s ataxia.

Reportedly, the research findings confirm the drug target of the HDAC inhibitors that Repligen is currently developing, for the treatment of inherited neurodegenerative diseases such as Friedreich’s ataxia.

The study entitled ‘Chemical Probes Identify a Role for Histone Deacetylase 3 in Friedreich’s Ataxia Gene Silencing’ was conducted in collaboration with scientists at The Scripps Research Institute.

Preclinical studies have shown that specific HDAC inhibitors increase production of the protein frataxin, which may have the potential to arrest disease progression in patients with Friedreich’s ataxia. Several potential clinical candidates synthesized by Repligen are completing characterization in preclinical models to identify the compound with the appropriate pharmacologic, toxicologic and pharmacodynamic profile for human clinical trials.

Repligen licensed the exclusive rights to intellectual property covering HDAC inhibitors from the Scripps Research Institute in April 2007, and the research efforts have been partially funded with grants from the Muscular Dystrophy Association, the Friedreich’s Ataxia Research Alliance, and the National Ataxia Foundation, said the company.

Walther Herlihy, president and CEO of Repligen, said: “Prior research indicated that HDAC enzymes play an important role in silencing the gene implicated in Friedreich’s ataxia. Identification of the involvement of HDAC 3 is an important step in developing a specific drug for Friedreich’s ataxia without the potential toxicities associated with broad-acting HDAC inhibitors. There are more than 15,000 patients worldwide with Friedreich’s ataxia with no therapies available for treatment.”

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.