CAMBRIDGE, Mass., Nov. 29, 2016 (GLOBE NEWSWIRE) -- Dimension Therapeutics, Inc. (NASDAQ:DMTX), a biopharmaceutical company advancing novel, adeno-associated virus (AAV) gene therapies targeting the liver, a key organ for human metabolism, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending DTX401 for designation as an orphan medicinal product for the treatment of Glycogen Storage Disease Type Ia (GSDIa). The positive opinion for DTX401 is a key milestone for Dimension's leading and unique program advancing the opportunity for AAV gene therapy for patients with limited treatment options to manage their disease. Designed to address the underlying genetic defect, DTX401 has been shown in multiple in vivo preclinical models to deliver stable expression and activity of the enzyme glucose-6-phosphatase-a (G6Pase-a). DTX401 has also been shown to improve G6Pase-a activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression. Dimension has completed candidate selection for its DTX401 program and is currently in IND-enabling studies. "We are pleased with the progress of our DTX401 program, which has followed a research pathway that includes studies in highly predictive preclinical in vivo models, application of well-described biomarkers, and the development of a proprietary manufacturing platform," said Dr. Annalisa Jenkins, MBBS, FRCP, Chief Executive Officer of Dimension. "In our current IND-enabling studies, we are replicating the groundbreaking work of leading researchers, particularly through our cooperative research and development agreement, or CRADA, with Janice Chou, Ph.D., at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at the National Institutes of Health (NIH)." David A. Weinstein, MD, who is joining Connecticut's UConn School of Medicine and Connecticut Children's Medical Center in early 2017 and is the chair of Dimension's GSDIa clinical advisory board, added, "The COMP positive opinion in the EU is an important milestone as it represents a key step forward for a potential treatment for GSDIa. Coupled with recent orphan drug designation for DTX401 in the US, the opinion affirms the substantial need for patients with GSDIa, who currently have limited therapeutic options. Affected individuals are at high risk for hypoglycemia (low blood sugar), coma, and potentially death, and are required to adhere to burdensome dietary regimens in order to maintain blood glucose at an appropriate level. Gene therapy is designed to address these challenges and offer a new option to patients."