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EINDHOVEN, THE NETHERLANDS
- Royal 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.”
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Schematic representation of the ultrasound technique called
sonoporation that will be researched by Philips and GlyGenix
Therapeutics for the targeted delivery of genes*.
Sonoporation
involves the use of microbubbles that are co-injected into the
bloodstream along with the therapeutic genes. When they arrive
at the target organ, the microbubbles are subjected to
high-intensity focused ultrasound causing them to rupture. This
increases the permeability of the blood vessel wall and cell
walls in the underlying tissue and facilitates the local uptake
of the therapeutic genes.
*Please note that the various components have not been drawn to
scale. |
“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 patients’ 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.
For further information, please contact:
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