Science and Technology
GlyGenix Therapeutics, Inc. has utilized a combination of scientific and business expertise to create an entire program designed to treat Glycogen Storage Disease Type 1a, for which there is no cure. Utilizing the most advanced technical applications in the fields of molecular biology and targeted gene therapy, GlyGenix Therapeutics, Inc. is engaged in strategies to manufacture and deliver genes to cure metabolic disorders that are caused by single gene mutations, with a primary focus on glycogen storage diseases.
The focus of GlyGenix Therapeutics, Inc.'s technology is the development of gene therapy drugs for the treatment of genetic disorders and is currently evaluating 2 different DNA-based drug formulations of an engineered glucose-6-phosphatase (G6Pase) gene for the treatment of GSD1a.
Current pre-clinical research in both a murine and a canine model of GSD1a demonstrate that the successful delivery of the G6Pase gene via adeno-associated virus (AAV) ameliorates disease symptoms. This has been shown to be curative, restoring normal glucose levels, growth and normalization of cholesteral, triglycerides and uric acid levels. In addition, glycogen deposits were significantly reduced resulting in the reduction of liver and kidney hypertrophy.