Spotlight Innovation announced that it has obtained an exclusive, worldwide license from Indiana University Research and Technology Corp. to commercialize STL-182, an orally-available small molecule that may have therapeutic potential for treating Spinal Muscular Atrophy (SMA). SMA is an autosomal recessive disorder that is a leading genetic cause of death in infants and toddlers. Synthesis and early preclinical testing of STL-182 was accomplished through a research collaboration between Professors Elliot Androphy of Indiana University School of Medicine and Kevin Hodgetts, director of the Laboratory for Drug Discovery in Neurodegeneration at Brigham and Women's Hospital. Their work was supported in part by the National Institute of Neurological Disorders and Stroke (NINDS) and the National Institute of Child Health and Human Development (NICHD).
Spinal Muscular Atrophy affects between 1 in 6,000 and 1 in 10,000 newborns. Approximately 1 in 40 to 1 in 50 adults have only a single intact spinal motor neuron 1 (SMN1) gene, which encodes a protein (SMN) required for proper neuromuscular function. An infant who inherits no intact SMN1 gene from either parent may develop SMA and lose the ability to sit, stand, walk, swallow, and/or breathe. In about 60% of cases, patients with SMA die by age two.
Even in SMA patients, low levels of functional SMN protein are produced by an SMN1-related gene called SMN2. One therapeutic strategy to treat SMA is to increase levels of functional SMN protein encoded by SMN2. In mouse models of SMA, STL-182 may restore neuromuscular function by stabilizing endogenous SMN protein.
Spinal Muscular Atrophy affects between 1 in 6,000 and 1 in 10,000 newborns. Approximately 1 in 40 to 1 in 50 adults have only a single intact spinal motor neuron 1 (SMN1) gene, which encodes a protein (SMN) required for proper neuromuscular function. An infant who inherits no intact SMN1 gene from either parent may develop SMA and lose the ability to sit, stand, walk, swallow, and/or breathe. In about 60% of cases, patients with SMA die by age two.
Even in SMA patients, low levels of functional SMN protein are produced by an SMN1-related gene called SMN2. One therapeutic strategy to treat SMA is to increase levels of functional SMN protein encoded by SMN2. In mouse models of SMA, STL-182 may restore neuromuscular function by stabilizing endogenous SMN protein.
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