Neurogene announces peer-reviewed publication of EXACT transgene technology

The company states: “Neurogene (NGNE) announced a peer-reviewed publication on its EXACT transgene regulation technology. The preclinical studies demonstrate EXACT’s ability to regulate gene therapy expression to a tolerable and therapeutic level, including in preclinical models of Rett syndrome. These data, published in Science Translational Medicine, supported the initiation of Neurogene’s ongoing Phase 1/2 clinical trial with NGN-401 gene therapy for Rett syndrome. Rett syndrome is caused by loss of function variants in the MECP2 gene; however, high levels of MeCP2 can lead to toxicity. Conventional, unregulated gene therapy leads to variable cellular uptake that can result in toxic levels of transgene expression. EXACT is a self-regulating circuit contained within a gene therapy cassette and enables therapeutic and tolerable transgene expression on a cell-by-cell basis through regulation by microRNA.”