Curis Awarded $5.4M to Identify Treatment for Spinal Muscular Atrophy

Curis Inc. in Cambridge has received a $5.4 million, three-year grant from the Spinal Muscular Atrophy Foundation to identify therapeutic compounds to treat spinal muscular atrophy, a debilitating neuromuscular disease that is the leading genetic cause of infant and toddler death.

The study will use proprietary Curis technologies and expertise to develop and refine motor neuron assays and to use those assays to screen for potential drug candidates.

Spinal muscular atrophy (SMA) is a genetic motor neuron disease caused by progressive degeneration of nerve cells in the spinal cord and brainstem, leading to muscle weakness, respiratory complications and premature death.

According to the National Institutes of Health, it is the most severe of the common childhood genetic neurological diseases and is the leading genetic cause of infant death. The SMA Foundation estimates that there are currently 25,000 to 55,000 people suffering from SMA in the United States, Europe and Japan.

“SMA is caused by decreased amounts of a specific protein that is necessary for survival of motor neurons,” said Lee Rubin, Curis’ chief scientific officer, in a statement. “Because of Curis’ unique ability to direct the differentiation of uncommitted cells into motor neurons, it has now become possible to screen for drug candidates in exactly the type of cell that is affected in the disease.”

The SMA Foundation is a nonprofit organization founded in 2003 and dedicated to finding a treatment and potential cure for SMA. The foundation is committed to raising awareness, education and increased federal funding and support.

Curis stock opened the day at $4.50, up 26 cents from its close last week.