“Our team continues to lead the industry in the development of a new, potentially disease-modifying therapeutic option for GM1 gangliosidosis. We are extremely proud of these data, representing our broadest dataset generated thus far, which support a dose response and a favorable safety and tolerability profile at both low and high doses. We observed dose-dependent responses in two key biomarkers, serum ß-galactosidase and cerebrospinal fluid (CSF) GM1 ganglioside, including normalization of both biomarkers in the high-dose cohort. Taken together, six out of seven patients show no evidence of overt disease progression at the latest timepoint assessed, and we now have a better understanding of the clinical measures that may serve as important indicators of efficacy. Based on the results of this ongoing study, we are working on the continued development of AXO-AAV-GM1 and plan to engage the FDA to discuss further development, recognizing that there is currently no approved therapy available for GM1 patients.”
“I have dedicated my career to the care of patients with GM1 gangliosidosis and to research efforts in the search for a functional cure. The biomarker dose response and favorable safety profile is a remarkable finding for the gene therapy field. I am excited about the potential impact that AXO-AAV-GM1 may have on the lives of these children and their families. I look forward to seeing the longer-term data, where we may have a chance to see not only durable disease stabilization, but possibly even improvement.”
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