Royal Philips Electronics and GlyGenix Therapeutics of Woodbridge, Conn., have formed a joint research agreement to explore the feasibility of using ultrasound technologies for gene therapy.
In particular, the collaboration will research the treatment of Glycogen Storage Disease Type 1a (GSD-1a) in pre-clinical studies. The collaboration will rely on Philips' medical imaging technologies for diagnosis and minimally-invasive medical procedures and GlyGenix's experience in correcting the genetic defect in GSD-1a.
GSD-1a is an inherited disease that makes it impossible for the body to regulate blood glucose levels, due to a defective G6Pase gene that prevents the body from producing an enzyme called glucose-6-phosphatase. Although it is a rare disease, affecting around one in every 100,000 to 200,000 births in the United States, it results in a significant reduction in quality of life and can lead to potentially life-threatening co-morbidities in early adulthood. Currently, there are no approved curative treatments for GSD-1a.
By directing focused ultrasound to target organs following DNA delivery, an increase in uptake--via a process known as sonoporation--has been successfully demonstrated in pre-clinical studies, according to the companies. Sonoporation increases the permeability of cell walls to allow the uptake of large molecules, thereby enabling the delivery of therapeutic genes. The proposed treatment is known as ultrasound-mediated plasmid DNA (pDNA) delivery.
The companies said their research program will target the expression of a functional human G6Pase therapeutic pDNA to the liver. Pre-clinical studies investigating its feasibility will be carried out by Philips and GlyGenix, in collaboration with the Duke University School of Medicine's division of medical genetics in Durham, N.C.