Neurogene (NASDAQ:NGNE) has announced an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) annual meeting this week describing a monitoring and treatment algorithm intended to reverse the rare, severe hyperinflammatory syndrome hemophagocytic lymphohistiocytosis (HLH) that has been associated with systemic exposure to high doses of adeno-associated virus (AAV) gene therapy (>1E14 vg/kg).
According to Neurogene, early monitoring and prompt treatment have proven effective in reversing the course of HLH in the setting of higher-dose AAV gene therapy (>1E14 vg/kg).
In a statement, Rachel McMinn, Ph.D., founder and CEO of Neurogene, said, “We appreciate the opportunity that ASGCT is providing for us to share findings related to HLH and the evidence-based recommendations for how to monitor, detect and treat this rare, hyperinflammatory syndrome in the context of AAV gene therapy. As we connect with the gene therapy community to provide this information, our hope is that it spurs adoption of the early monitoring and treatment algorithm and increases propensity for sharing trial findings from which others can learn.”
