Our team at Nationwide Children’s Hospital has continued progress toward initiating clinical trials of gene therapy in MPSIIIA and MPSIIIB. The team has completed 12-month visits for the 25 subjects enrolled in the MPSIII Natural History Study. This study, which was supported in part by The Children’s Medical Research Foundation, was designed to evaluate potential outcome measures for therapeutic trials, and to assess how these measures change in individual patients over time. Preliminary results have been presented at meetings of the American College of Medical Genetics in Salt Lake City in March, 2015 and the American Society of Human Genetics in October, 2015. The NCH team is currently preparing a manuscript for publication that will present the results to the MPS worldwide research community, and we will present results of the study at the WORLD symposium in San Diego in March, 2016.
Critical data from this study was also included in an Investigational New Drug (IND) application that we have submitted to the FDA requesting approval to perform a clinical trial of gene transfer for MPSIIIB. While we are still awaiting the formal response from the FDA we are proceeding with the preparation of an IND submission for the MPSIIIA clinical trial as well. Both gene transfer trials await completion of production and final release of the necessary viral vector.