NIH Grant to Accelerate Development of Innovative Non-Viral,
Non-Lipid Platform Originating from Cincinnati Children's
CINCINNATI, July 30,
2024 /PRNewswire/ -- Genexys BioMed, a
pre-clinical stage biotechnology company developing a novel
non-viral, non-lipid genetic medicine platform for the treatment of
cystic fibrosis (CF) and other genetic diseases, announced today it
has been awarded a $320,000 Phase 1
Small Business Innovation Research (SBIR) grant from the National
Heart, Lung, and Blood Institute (NHLBI) at the National Institutes
of Health (NIH).
The SBIR award will enable Genexys BioMed to advance its
innovative approach of delivering therapeutic mRNA or DNA directly
to CF patients' lungs using a non-viral delivery system. This
approach aims to overcome the challenges with current viral-based
and lipid-based gene therapies, offering a safer and potentially
more effective treatment option for patients suffering from this
devastating genetic disorder.
Cystic fibrosis is a serious genetic disorder affecting around
100,000 people worldwide, including about 40,000 in the United States. CF mainly impacts the lungs
and digestive system and is caused by mutations in the CFTR
gene, with about 70% of CF patients carrying the delta F508
(F508del) mutation. Current therapies primarily target this common
mutation, leaving a significant portion of CF patients with no
treatment options. Genexys BioMed's non-viral gene therapy platform
is designed to be effective for all CF patients, regardless of
their specific genetic mutation, and also has potential
applications for other genetic diseases.
"This SBIR grant is a significant milestone for Genexys BioMed
and provides validation for our groundbreaking non-viral gene
therapy platform," said Peter Alff,
Ph.D., CEO of Genexys BioMed and Senior Venture Partner at Orange
Grove Bio. "Our versatile approach has the potential to overcome
long standing challenges in cystic fibrosis treatment, while also
opening up possibilities for treating a wide range of genetic
diseases."
Genexys BioMed's gene therapy technology was advanced at
Cincinnati Children's in the lab of Dr. Assem Ziady, an expert in CF research and gene
therapy. By leveraging a highly modular organic polymer-based
platform and enhancers of gene transfer, Genexys BioMed's
technology can scalably deliver large genetic payloads while
minimizing immunogenicity and toxicity. The approach also enables
precise targeting of specific cell types making the platform
potentially useful across a wide range of therapeutic
applications.
"Gene therapy holds immense promise for cystic fibrosis, but its
clinical implementation has been impeded by the lack of safe and
efficient delivery vehicles," said Dr. Colin Sheehan, Genexys BioMed's Senior Scientist
and lead investigator in the SBIR award. "Our non-viral delivery
approach aims to overcome these hurdles, potentially improving
outcomes for all CF patients, irrespective of their CFTR mutation,
and offering new hope to affected patients and families."
"We are excited to see the progress of Genexys BioMed in
advancing our gene therapy approach and platform for CF," said Dr.
Ziady, professor at Cincinnati Children's and a lead inventor of
targeted and enhanced nucleic acid nanoparticles. "This
collaboration between academia and industry exemplifies how
research can be translated into therapies for patients. We look
forward to Genexys BioMed bringing this promising technology closer
to clinical application."
About Genexys BioMed
Genexys BioMed, a subsidiary of Orange Grove Bio, is a
pre-clinical biotechnology company focused on developing a novel,
highly modular non-viral, non-lipid gene therapy platform for the
treatment of cystic fibrosis (CF). Our innovative gene therapy
platform aims to address the significant unmet medical need in CF
treatment, offering advantages over traditional viral and
lipid-based methods. Our approach has shown promising results in
both in vitro and in vivo studies, focusing on optimizing the
delivery and functionality of the CFTR gene. For more information
about Genexys BioMed and our pioneering gene therapy platform,
please visit www.orangegrovebio.com.
Media Contacts:
Chris Hempel
Spark Public Relations
chris.hempel@sparkpr.com
+1-775-813-0285
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SOURCE Genexys BioMed