Leading the Way in Life Science Technologies

GEN Exclusives

More »

GEN News Highlights

More »
Apr 11, 2007

Repligen Licenses Potential Treatment for Friedreich's Ataxia

  • Repligen entered into an exclusive commercial license with The Scripps Research Institute for intellectual property covering compounds that may have utility in treating Friedreich's ataxia.

    Friedreich's ataxia is an inherited neurodegenerative disease in which low levels of the protein frataxin result in progressive damage to the nervous system and loss of muscle function.

    According to the company, research in tissues derived from patients as well as in mice indicates that these compounds increase production of the protein frataxin, low levels of which result in progressive damage to the nervous system and loss of muscle function.

    There is currently no treatment or cure for Friedreich's ataxia, Repligen adds. These compounds are the only ones to date that have demonstrated utility in increasing both the level of the frataxin protein in tissue samples from patients with Friedreich's ataxia as well as frataxin gene activity in animal models, the company says.

    Repligen also reports that preliminary data suggests that these compounds may have utility in treating other disorders, such as spinal muscular atrophy and Huntington's disease.



Be sure to take the GEN Poll

Cancer vs. Zika: What Worries You Most?

While Zika continues to garner a lot of news coverage, a Mayo Clinic survey reveals that Americans believe the country’s most significant healthcare challenge is cancer. Compared to other diseases, does the possibility of developing cancer worry you the most?

More »