Solve GNE and Genosera Sign Agreement to Develop a Novel Bicistronic AAV Gene Therapy Targeting HIBM


LOS ANGELES, Aug. 14, 2023 (GLOBE NEWSWIRE) -- Solve GNE announced today that it has entered into a sponsored research agreement with Genosera to provide funding for a novel Adeno-Associated Virus (AAV) gene therapy aimed at curing GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM). GNE Myopathy is a genetic disorder characterized by muscle weakness that starts in early adulthood. The progression of the disease varies among patients but generally starts in the leg muscles and eventually leads to complete loss of mobility. Although this is a rare disorder, its incidence is remarkably high among Iranians of Jewish decent, where it’s estimated that 10-15% of the population carry at least a single copy of the M743T point mutation.

Gidon Akler, MD, and Paul T. Martin, PhD, co-founded Genosera in 2019 with the goal of developing new gene therapy treatments for patients with rare genetic disorders. The company has a particular focus on genetic diseases that are common in members of the Iranian Jewish community, including GNE myopathy and Lysosomal Acid Lipase Deficiency. Genosera is developing novel bicistronic, or two gene, AAV treatments for muscle diseases. Such therapies will, for the first time, provide the possibility of stopping disease progression and also reversing pre-existing muscle disease. Together with Solve GNE, Genosera will work to develop the first bicistronic gene therapy treatment for GNE myopathy patients. Genosera’s product pipeline comes from research done in Dr. Martin’s laboratory in the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital in Columbus Ohio.

Alexander Monsef of Solve GNE commented, “The Center for Gene Therapy at Nationwide Children’s Hospital (NCH) has a distinguished record of research in gene therapy and its scientists have played a major role in the development of landmark therapies like Zolgensma for Spinal Muscular Atrophy, and Elevidys for Duchenne Muscular Dystrophy. Solve GNE is proud to be collaborating with Genosera and NCH towards finding a cure for GNEM.”

About Solve GNE
Solve GNE is a Los Angeles based non-profit organization focused on developing a cure for GNE Myopathy (or HIBM). Solve GNE has been actively raising funds to support research and development of various gene therapies that are poised to enter human clinical trials in the next 12-18 months. For more information, visit www.solvegne.org

About Genosera
Founded in 2019, Genosera is a privately held, gene therapy biotechnology company that focuses on various rare genetic diseases such as GNEM, Lysosomal Acid Lipase Deficiency (LAL-D) and Limb-girdle muscular dystrophy type 2I (LGMD2I). For more information, visit www.genosera.com

Forward-Looking Statements
This press release contains forward-looking statements, including, without limitation, statements regarding the success, cost, and timing of our product development activities and clinical trials, our plans to research, develop, and commercialize our product candidates, and our plans to submit regulatory filings and obtain regulatory approval of our product candidates. These forward-looking statements are based on Genosera’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks, and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include but are not limited to: (a) the timing, costs, and outcomes of our clinical trials and preclinical studies, (b) the timing and likelihood of regulatory filings and approvals for our product candidates, and (c) the potential market size for our product candidates. These forward-looking statements speak only as of the date made and, other than as required by law, we undertake no obligation to publicly update or revise any forward-looking statements. This press release does not constitute an offer to sell, or a solicitation of an offer to buy, any securities.



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