Fortress Biotech, Cyprium Therapeutics announces $4.1M grant from NINDS

Cyprium Therapeutics, a majority-owned subsidiary of Fortress Biotech, announced that the National Institute of Neurological Disorders and Stroke, NINDS, of the National Institutes of Health, NIH, has awarded a three-year grant totaling approximately $4.1M to the Research Institute at Nationwide Children’s Hospital and Principal Investigator, Stephen G. Kaler, M.D., M.P.H., to fund completion of preclinical studies, manufacturing and preparation of an Investigational New Drug Application for a first-in-human clinical trial to advance adeno-associated virus-ATP7A gene therapy, also known as AAV-ATP7A, for the treatment of Menkes disease. Lindsay Rosenwald, M.D., Executive Chairman, President and Chief Executive Officer of Fortress, stated, “We are honored to have the NIH’s support, and Dr. Kaler’s and Nationwide Children’s leadership, to progress the development of AAV-ATP7A gene therapy, to help provide a more effective therapeutic option to patients with Menkes disease. As we continue the research and development of AAV-ATP7A in combination with CUTX-101, there is great potential for this next generation approach to enhance the treatment of Menkes disease, which currently has no FDA-approved treatment. We look forward to continuing to advance AAV-ATP7A with Dr. Kaler and supporting Sentynl as they finalize and complete the NDA submission for CUTX-101.”