Enzo Biochem, Inc. and the Spinal Muscular Atrophy (SMA) Foundation today announced that Enzo's wholly owned subsidiary, Enzo Life Sciences Inc., has launched a unique immunoassay (ELISA) system which can be used for the identification and detection of Survival Motor Neuron (SMN) protein. The kit is the result of a collaborative agreement between Enzo and the SMA Foundation for the development of reagents and assays for SMN protein. The availability of an effective SMN ELISA could further enable and expedite drug discovery, development and therapy for Spinal Muscular Atrophy, the leading genetic cause of mortality in infants and toddlers.
SMA is a genetic, neuromuscular disease caused by progressive degeneration of nerve cells in the spinal cord that leads to muscular weakness and atrophy and increased risk for early death due to respiratory failure. It is estimated that SMA affects approximately 1 in 6,000 to 1 in 10,000 live births worldwide. The disease is linked to a mutation in or deletion of the SMN1 gene, resulting in reduced levels of SMN protein. An active area of therapeutics development has been elevation of SMN protein levels.
"The SMN ELISA kit addresses a critical gap in SMA research and is expected to significantly accelerate SMA therapeutics development. Development and distribution of this system could not happen without the successful collaboration with Enzo and contributions from the SMA research community," said Karen Chen, Ph.D., the Chief Scientific Officer of the SMA Foundation.
The Enzo SMN ELISA Kit provides a reliable and widely-accessible means for researchers to measure SMN protein levels, and greatly simplifies and accelerates efficacy assessment of potential drugs in clinical trials that are designed to increase SMN protein levels. The assay has been validated for use with human and mouse cell lysates and tissue homogenates, such as peripheral blood mononuclear cell (PBMC) lysates, and will be marketed through Enzo's worldwide sales and marketing group.
"During the assay development, the SMA Foundation facilitated the supply of assay kits to affiliated researchers in the US and abroad. This allowed us to validate the assay in-house while simultaneously receiving valuable feedback about assay specifications, performance, and protocols from scientists who will ultimately be using the kits in their studies," said Wayne Patton Ph.D., Chief Scientific Officer at Enzo Life Sciences. "Our custom assay development team continues to work with the SMA Foundation to expand the utility of the kit to include additional sample types and protocols relevant to SMA research programs and model systems, which should add further value to the assay in the marketplace."