The FDA has granted ExSAR Corporation Orphan-Drug Designation for investigational drug N-Acetyl-Glucosamine Thiazoline (NGT) for the treatment of adult Tay-Sachs disease.

Adult (or Late-Onset) Tay Sachs affects adults rather than infants, and manifests itself as a progressive loss of functioning of the nervous system. Tay-Sachs disease is caused by the absence of a vital enzyme called hexosaminidase A (Hex-A). Without Hex-A, a fatty substance or lipid called GM2 ganglioside accumulates abnormally in cells, especially in the nerve cells of the brain. This ongoing accumulation causes progressive damage to the cells. The enzyme defect, is the same as that of classical Tay-Sachs, but people with the late-onset condition have significantly reduced amounts of Hex-A rather than a complete absence of the enzyme.

In late 2005 ExSAR licensed NGT from The Hospital for Sick Children in Toronto, Canada. NGT is a small molecule which acts as a pharmacological chaperone to assist proper folding of the Hex-A, thereby allowing its transport out of the endoplasmic reticulum into the lysosome where it digests GM2 ganglioside in neurons.

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