Genexys BioMed Awarded $320K SBIR Grant to Advance Novel Cystic Fibrosis Gene Therapy

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.

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SOURCE Genexys BioMed