Welcome to GlyGenix Therapeutics, Inc.
Focused on Treating Genetic Diseases
GlyGenix Therapeutics, Inc. (GTI) 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.
We are currently working with several leading research institutions and scientists in the fields of gene therapy and GSD.
Dr. David Weinstein, the Director of The Glycogen Storage Disease Program at the University of Florida, is excited to be working with GlyGenix Therapeutics, Inc. on the effort to get gene therapy treatment into humans with GSDIa. "With GTI as an industry partner, the chance of moving this treatment forward in a timely manner substantially increases. In addition, GlyGenix is critical for helping to obtain the funding required for manufacturing the viral vector and the company will be instrumental at helping to get FDA approval for the future gene therapy trial in GSDI."
As the Director of the PGTC Toxicology Center, Dr. Thomas Conlon adds, "Our gene therapy group at the University of Florida has been working with GlyGenix Therapeutics, Inc. over the course of the last year and a half to develop a strategy and clinical road map for developing a gene therapy drug candidate for GSD1a. Our expertise and the efforts from GTI to provide the stimulus and excitement for finding a cure for this disease and provides the team effort approach that is needed to develop new therapies for orphan diseases, such as GSD1a."
On March 11, 2013, the FDA approved Orphan Drug Designation for our lead candidate drug, AAV-G6Pase.
- 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.