Tay-Sachs Disease is a rare inherited disorder that progressively destroys nerve cells in the brain and spinal cord. Sandhoff disease, also known as Sandhoff–Jatzkewitz disease, variant 0 of GM2-Gangliosidosis or Hexosaminidase A and B deficiency, is a lysosomal genetic, lipid storage disorder caused by the inherited deficiency to create functional beta-hexosaminidases A and B. These catabolic enzymes are needed to degrade the neuronal membrane components, ganglioside GM2, its derivative GA2, the glycolipid globoside in visceral tissues, and some oligosaccharides. Accumulation of these metabolites leads to a progressive destruction of the central nervous system and eventually to death.
IntraBio Inc., a late-stage biopharmaceutical company, today announced that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) Application for Clinical Trial IB1001-202 involving its lead compound (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).
The IND approval of IB1001-202 allows IntraBio to move forward with the trial at U.S. clinical sites. In addition to the U.S. centers, IntraBio intends to commence the study in European countries, including the United Kingdom, Germany, and Spain.Source: Pharmiweb