Repligen Corporation has entered an exclusive agreement with Families of Spinal Muscular Atrophy (FSMA) to license compounds with the potential to treat spinal muscular atrophy (SMA).
SMA is an as-yet incurable inherited neurodegenerative disease in which a gene defect results in low levels of the protein SMN, leading to progressive damage to motor neurons, loss of muscle function and death.
The licensed compounds snapped up by Repligen increase the production of SMN in cells derived from patients. The treatments have demonstrated significantly increased survival rates in animal studies.
Repligen Corporation president and chief executive officer Walter C Herlihy said that FSMA had made remarkable progress in defining compounds that may be clinical candidates for SMA.
"We look forward to working with FSMA and their collaborators in the development of what we hope will be an important new treatment for SMA," Herlihy said.
SMA is diagnosed in approximately one in 6,000 in the US and Europe, where about 20,000 patients are estimated to suffer from the disease.