N-acetylmannosamine (ManNAc) is a small uncharged physiological molecule that crosses membranes readily and is the natural precursor of intracellular sialic acid synthesis. NHGRI investigators discovered that ManNAc can be used for therapeutic purposes, including treating certain kidney diseases (e.g., those involving abnormal levels of protein in the urine and/or blood in the urine), resulting primarily or secondarily from hyposialylation (deficiency of sialic acid). Notably, ManNAc can also potentially be used to treat diabetic nephropathy.
ManNAc therapy is given orally and shows long-term safety and biochemical efficacy, consistent with its mechanism of action in humans.
Thus, the following fields of use are available for licensing: Treating kidney disorders due to hyposialylation of the glomerular basement membrane (GBM), including but not limited to minimal change disease glomerulopathy (MCD), focal segmental glomerulosclerosis (FSGS), membranous nephropathy (MN), and diabetic nephropathy in humans with oral formulations of N-acetyl mannosamine (ManNAc) or derivative.