Philips and GlyGenix Therapeutics Team up to Research Ultrasound-Mediated Gene Therapy

Royal Philips ElectronicsRoyal Philips Electronics (NYSE: PHG, AEX: PHI) and GlyGenix Therapeutics, Inc. (Woodbridge, Connecticut, USA) today announced 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 unites Philips' expertise in medical imaging technologies for diagnosis and minimally-invasive medical procedures with GlyGenix's expertise in correcting the genetic defect in GSD-1a.

"The potential to deliver genes using a targeted approach will be a significant advance for correcting genetic defects and could offer the prospect of curing hereditary diseases such as GSD-1a," commented William Fodor, CSO of GlyGenix Therapeutics, Inc. "Philips' ultrasound-mediated DNA delivery techniques offer the opportunity to deliver genes without the size constraints and limitations of viral packaging systems, and thus open the door to the development of more robust and effective therapeutic genes."

"Medical imaging systems already play a crucial role in minimally-invasive medical procedures such as opening obstructed arteries, correcting heart rhythm disorders, or sampling tissue biopsies of suspected lesions," said Henk van Houten, senior vice president of Philips Research and head of the Healthcare research program. "The development of ultrasound techniques that could non-invasively target the delivery of drugs, genes and stem cells to specific parts of the body opens up further possibilities to advance patient care."

GSD-1a is an inherited disease that makes it impossible for the body to regulate blood sugar (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, only affecting around 1 in every 100,000 to 200,000 births in the USA, it results in a significant reduction in patientsÂ’ 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. Correcting the genetic defect that causes it could offer the prospect of an effective therapy that would allow patients with GSD-1a to lead a normal life.

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.

Related news articles:

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.

About GlyGenix Therapeutics
Based in Woodbridge, CT, GlyGenix Therapeutics, Inc. is a privately held biotech company developing therapeutic solutions for severe metabolic disorders. The company's initial focus is in using gene therapy products with non-viral delivery systems for the treatment of Glycogen Storage Disease Type 1a (GSD1a), a rare and severe chronic genetic liver disease for which no approved therapies exist. Founded in 2006, and with the aid of a sizable body of pre-clinical data, GlyGenix Therapeutics, Inc is poised to expedite the initiation of clinical trials for the treatment of GSD1a. News from GlyGenix Therapeutics, Inc is located at www.glygenixtherapeutics.com.

Most Popular Now

Using Data and AI to Create Better Healt…

Academic medical centers could transform patient care by adopting principles from learning health systems principles, according to researchers from Weill Cornell Medicine and the University of California, San Diego. In...

AI Medical Receptionist Modernizing Doct…

A virtual medical receptionist named "Cassie," developed through research at Texas A&M University, is transforming the way patients interact with health care providers. Cassie is a digital-human assistant created by Humanate...

Northern Ireland Completes Nationwide Ro…

Go-lives at Western and Southern health and social care trusts mean every pathology service is using the same laboratory information management system; improving efficiency and quality. An ambitious technology project to...

AI Tool Set to Transform Characterisatio…

A multinational team of researchers, co-led by the Garvan Institute of Medical Research, has developed and tested a new AI tool to better characterise the diversity of individual cells within...

AI Detects Hidden Heart Disease Using Ex…

Mass General Brigham researchers have developed a new AI tool in collaboration with the United States Department of Veterans Affairs (VA) to probe through previously collected CT scans and identify...

Human-AI Collectives Make the Most Accur…

Diagnostic errors are among the most serious problems in everyday medical practice. AI systems - especially large language models (LLMs) like ChatGPT-4, Gemini, or Claude 3 - offer new ways...

Highland Marketing Announced as Official…

Highland Marketing has been named, for the second year running, the official communications partner for HETT Show 2025, the UK's leading digital health conference and exhibition. Taking place 7-8 October...

MHP-Net: A Revolutionary AI Model for Ac…

Liver cancer is the sixth most common cancer globally and a leading cause of cancer-related deaths. Accurate segmentation of liver tumors is a crucial step for the management of the...

Groundbreaking TACIT Algorithm Offers Ne…

Researchers at VCU Massey Comprehensive Cancer Center have developed a novel algorithm that could provide a revolutionary tool for determining the best options for patients - both in the treatment...

The Many Ways that AI Enters Rheumatolog…

High-resolution computed tomography (HRCT) is the standard to diagnose and assess progression in interstitial lung disease (ILD), a key feature in systemic sclerosis (SSc). But AI-assisted interpretation has the potential...