Azafaros has announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102 to treat GM2 gangliosidosis, including Tay-Sachs disease and Sandhoff disease. GM2 gangliosidosis, a group of rare genetic disorders that progressively destroy nerves in the brain and spinal cord, is sometimes referred to as Tay-Sachs or Sandhoff disease and is characterized by a deficiency of the enzyme beta-hexosaminidase. GM2 gangliosidosis mainly disrupts the normal activity of lysosomes within cells and is most common and severe in infants and children. The ODD was granted for, AZ-3102, an orally ingested small molecule azasugar that selectivity inhibits glucosylceramide synthase (GCS) and non-lysosomal neutral glucosylceramidase (GbA2), two enzymes involved in glycolipid metabolism. Currently, there is no treatment for GM2 gangliosidosis but several therapies exist for managing symptoms. Azafaros was founded in 2018 and develops disease-modifying therapeutics for rare genetic conditions.
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