Welcome to GlyGenix Therapeutics, Inc.

Focused on restoring metabolism

GlyGenix Therapeutics, Inc. is focused on treating and curing Glycogen Storage Diseases (GSDs). Our primary focus is to cure GSD type 1a, which is caused by an inherited gene mutation resulting in altered glycogen metabolism and glucose homeostasis. We are currently evaluating several in vivo approaches for targeting the normal gene to the appropriate tissue, including Adeno-Associated Viral (AAV) vector mediated gene therapy as well as non-viral delivery approaches. The product opportunities target unmet clinical needs and are expected to represent a significant therapeutic advance. GlyGenix Therapeutics, Inc.'s mission is focused on the development of nucleic acid molecules that compensate or correct the genetic defects that cause Glycogen Storage Disease Type 1a.

GSD1a Facts

  • GSD1a is a genetic disorder with a prevalence of 1:100,000 live births.
  • Found in almost every culture around the world, it is estimated that 1:68 Ashkenazi Jews are carriers for GSD1a.
  • The liver enzyme responsible for blood glucose maintenance is either missing or dysfunctional, making it difficult to maintain normal glucose levels between meals.
  • There is no existing medication or cure for this disease.
  • Many children need to undergo intensive therapy to relearn sucking, swallowing and speech.