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Mitotherapeutix Awarded $2.2 Million SBIR Grant from the National Institutes of Health for siRNA NASH Candidates

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FARMINGTON, Conn. (PRWEB)

Mitotherapeutix LLC (MITO), a pre-clinical stage company focused on siRNA drug discovery and development based on suppression of the mitochondrial regulator MCJ (DnaJC15), today announced that it has been awarded a multi-year $2.2 million Small Business Innovation Research (SBIR) grant from the National Institutes of Health. MITO will deploy the funds to continue IND enabling activities for siRNA candidates intended to be a novel treatment for Nonalcoholic Steatohepatitis (NASH).

“NASH remains a significant unmet medical need affecting up to 5% of all adults in the United States, according to the Liver Foundation,” said Jack Talley, Chief Executive Officer, MITO. “Our novel GalNAc conjugated-siRNA candidates are being advanced towards IND filing in late 2022. The SBIR grant will provide non-dilutive funding for rodent and monkey toxicity studies as well as pharmacodynamic studies in monkeys that are key to successful IND filing.

“We developed the GalNAc delivery platform for targeting the liver and siRNA targeted MCJ in-house and are delighted with the support from NIH for our endeavors.”

The Company is seeking funding by way of commercial partnerships or the sale of equity in order to advance the MCJ platform forward.

About NASH:

The normal liver does not store fat, but fat can be found in individuals that have Nonalcoholic Fatty Liver Disease (NAFLD). If there is hepatitis (inflammation) in addition to fat accumulation, then the disease is recognized as Nonalcoholic Steatohepatitis (NASH). According to the National Institute of Diabetes and Digestive and Kidney Diseases (NDDK) as many as 30% to 40% of Americans have NAFLD and about 3% to 12% have NASH. Dietary changes and weight loss can be of major help in the treatment of NASH but occurs in only a small number of individuals. There is major activity in drug discovery and development for NAFLD and NASH. Thus far no drug has been approved. We believe our novel approach enhancing the metabolic activity in mitochondria has great potential. We do this by enhancing electron transport chain activity (ETC) in mitochondria and have demonstrated the efficacy of this approach in several animal models of NASH.

About Mitotherapeutix LLC:

Mitotherapeutix is a US-based pre-clinical stage pharmaceutical company that is developing drugs (siRNA) targeting metabolism to treat disease. Our initial program targets a protein MCJ/DNAJC15, found in the mitochondria and is responsible for enhancing the production of energy. MCJ is unusual because it is a negative regulator of metabolism and knocking out MCJ enhances the electron transport chain (ETC) and the production of ATP (called mitochondrial respiration) without increasing reactive oxygen species (ROS). Enhancing the capacity of mitochondria to burn the fat in the liver and generate ATP, reduces fat accumulation in the liver and, thereby, liver inflammation and fibrosis. MCJ is also present in other tissues besides the liver including the kidney and the heart. For more information, please visit http://mitotherapeutix.com.

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