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Marker Gene Gets $1.6M SBIR Grant to Build Out Screening Tech for Lysosomal Storage Disease

NEW YORK (GenomeWeb News) – Marker Gene Technologies today announced a three-year, $1.6 million grant from the National Institutes of Health to further the company's screening technology directed at drugs for lysosomal storage disease.

The Eugene, Ore.-based company will use the Phase II Small Business Innovation Research grant from the National Institute of Neurological Disorders and Stroke to develop live cell and tissue assays to screen drug candidates using fluorogenic, targeted substrate reporters of lysosomal enzyme activity.

According to the grant abstract, in Phase I work, MGT established the feasibility of its technology "by preparing new fluorogenic glycosidase, esterase, phosphatase, lipase and sulfatase substrates for lysosomal enzymes and demonstrated differential staining in living cells that were from normal or were of disease origin or upon induction of inhibition of lysosomal enzyme activities."

In the next phase of research funded by the Phase II grant, the new substrates will be assayed in vitro "for their ability to measure specific and localized inhibition or induction of lysosomal enzymes in living cells as well as differentiate individual enzyme activities in a cell- or tissue-specific manner."

MGT will also validate the new systems for use in high-throughput screening for drug discovery and for use in clinical diagnostics for evaluating the occurrence and progression of disease and monitoring the effectiveness of therapeutic treatments.

Lysosomal storage diseases are metabolic disorders of the nervous systems and include Tay-Sachs, Gaucher's, and Niemann-Pick disease. About one in every 100,000 people has a lysosomal storage disease, MGT said.

"The new substrates and the resulting detection systems will provide innovative methods to quantitate lysosomal enzyme function and to screen for the influence of secondary drug or protein administration, making them useful medical research tools for a variety of significant biochemical and medical applications," MGT President and CEO John Naleway said in a statement.

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