Scripps’ research has shown that compounds increase frataxin protein and gene activity.
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.