Genosera Inc. Plans Clinical Development with Novel Dual-gene Vector in Neuromuscular Diseases
Ohio Pen/10329048

COLUMBUS, Ohio - OhioPen -- COLUMBUS, Ohio, Genosera Inc., a pre-clinical stage gene therapy company, today announced that it has secured three exclusive licenses from Nationwide Children's Hospital covering seven intellectual property assets. The assets cover a platform of novel dual-gene AAV vectors that couple gene replacement therapy for genetic diseases with a second muscle growth/regeneration therapy to build new muscle mass and strength. This dual-gene AAV therapy approach enhances therapeutic efficacy compared to single gene replacement therapies currently approved for patients.

In pre-clinical studies, the dual gene AAV approach shows not just disease corrective actions but induction of restorative functions when muscle disease has already progressed.  Genosera's platform will change the paradigm for the treatment of genetic muscle diseases, providing the potential to not only stop disease progression but reverse disease in more advanced patients. This approach provides a potential curative therapy, restoring lost muscle mass and strength for neuromuscular disorders, utilizing much lower AAV doses than currently approved therapies.

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Genosera's therapeutic approach will be first assessed in two neuromuscular diseases where the FDA has not approved any therapies: GNE myopathy and LGMD2i (now called LGMDR9).  Patients with GNE myopathy have mutations in the GNE gene, the gene encoding a dual function enzyme that synthesizes the committed steps in sialic acid biosynthesis.  Patients with LGMDR9 have mutations in the FKRP (Fukutin-related protein) gene, which encodes an enzyme required for functional glycosylation of the muscle membrane.

"With our proprietary platform of novel AAV vectors, we have shown dramatic improvements, including reversal of symptoms, in disease models.  We anticipate meeting with the FDA this year to discuss plans for our clinical trials" says Dr. Patricia Beckmann, Chief Executive Officer.

"Further, we believe our dual-gene AAV platform has therapeutic applications in more than two dozen other neuromuscular diseases, as well as some far more common non-genetic conditions such as sarcopenia. By proving safety and efficacy in these first two rare genetic diseases, we can establish Genosera in what should be an expanding portfolio of disease treatments using this new technology."

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ABOUT GENOSERA INC.

Founded in 2019, in Columbus, Ohio, Genosera, Inc. is a privately held pre-clinical stage, gene therapy company.  Genosera has developed a pipeline of proprietary innovative dual-gene AAV gene therapy treatments for rare genetic diseases.  Their first-in-class AAV therapies provide a new platform for the treatment of neuromuscular and other disorders. For more information, please visit Genosera's website at www.genosera.com.