Repligen signs license deal with Scripps

In Friedreich’s ataxia, low levels of the protein frataxin result in progressive damage to the nervous system and loss of muscle function.

Research in tissues derived from patients as well as in mice indicates that the licensed compounds increase production of the protein frataxin, which suggests potential utility of these compounds in slowing or stopping progression of the disease.

There is currently no treatment or cure for Friedreich’s ataxia. Preliminary data also suggests that these compounds may have utility in treating other disorders such as spinal muscular atrophy and Huntington’s disease.