Current gene therapies that rely solely on the bloodstream to deliver corrective gene molecules typically fail to deliver sufficient quantities to the target organs. However, 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. Sonoporation increases the permeability of cell walls to allow the uptake of large molecules, thereby enabling the delivery of therapeutic genes.

Compared to current gene therapies that use viral vectors to infect cells, this ultrasound-mediated technique carries no risk of an anti-viral immune or inflammatory response. In addition, this targeted approach could reduce side effects.


The proposed treatment is known as ultrasound-mediated plasmid DNA (pDNA) delivery. The research program into it will specifically target the expression of a functional human G6Pase therapeutic pDNA to the liver, the primary organ responsible for glycogen storage and glucose release. Pre-clinical studies to investigate the feasibility of the technique will be carried out by Philips Research and GlyGenix Therapeutics in collaboration with the Duke University School of Medicine's Division of Medical Genetics (Durham, North Carolina, USA) - a recognized leader in GSD-1a diagnosis, managed care, pediatric genetics and experimental models.

GlyGenix Therapeutics, Inc. holds a worldwide exclusive license to the G6Pase gene, protein, and related mutations for the treatment of GSD-1a. GlyGenix will seek to obtain orphan drug designation for the treatment of GSD-1a, which would provide 7 years of market exclusivity.

"The latest collaboration with GlyGenix Therapeutics is part of a broader set of
activities which Philips has undertaken with the aim to bring image-guided drug
delivery to patients. These activities include an ongoing collaboration with Celsion Columbia, MD, USA) in oncology and leading the European SonoDrugs project, which is committed to developing novel drug delivery vehicles for image-guided drug delivery in oncology and cardiology," Dr. Pashley said. "Our collaboration with GlyGenix provides a good opportunity to demonstrate the ability to enhance gene uptake by the use of ultrasound in a relatively short period of time because it is easy to determine if the therapy has been successful."

About Royal Philips Electronics

Royal Philips Electronics of the Netherlands (NYSE: PHG, AEX: PHI) is a diversified Health and Well-being company, focused on improving people's lives through timely innovations. As a world leader in healthcare, lifestyle and lighting, Philips integrates technologies and design into people-centric solutions, based on fundamental customer insights and the brand promise of "sense and simplicity". Headquartered in the Netherlands, Philips employs approximately 116,000 employees in more than 60 countries worldwide. With sales of EUR 26 billion in 2008, the company is a market leader in cardiac care, acute care and home healthcare, energy efficient lighting solutions and new lighting applications, as well as lifestyle products for personal well-being and pleasure with strong leadership positions in flat TV, male shaving and grooming, portable entertainment and oral healthcare. News from Philips is located at www.philips.com/newscenter.

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