|Report Code: PP10107||Report Type: Indication Pipeline Reports||Available format:|
Tay-Sachs is a lysosomal storage disorder caused by the deficiency of vital enzymes called beta-hexosaminidase A (Hex-A). The role of Hex-A is to degrade a fatty substance or lipid called GM-2 ganglioside. In the absence of the Hex-A enzymes, GM-2 accumulates abnormally in cells, especially in the nerve cells or neurons, of the brain. In children, the destructive process begins in the fetus early in pregnancy. However, a baby with Tay-Sachs disease appears normal till six months of age when its develops slowly. By about two years of age, most children experience recurrent seizures and diminishing mental functioning. The infant gradually regresses, and is eventually unable to crawl, turn over, sit or reach out. There is no specific treatment for Tay-Sachs disease.
The drug candidates in Tay-Sachs therapeutics pipeline include, but not limited to, CCP-010. Some of the major companies having drugs in the Tay-Sachs therapeutics pipeline includes Lysogene, and Castle Creek Pharmaceuticals.