Neurogene Announces New Development Program in Rett Syndrome Utilizing Novel EXACT Technology Platform
Promising preclinical data for NGN-401 presented at 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) demonstrate significant survival benefit and reduced disease burden in Rett syndrome mouse model Results show Neurogene’s EXACT gene therapy regulates MECP2 gene expression to target levels, avoiding overexpression related toxicity EXACT platform holds potential to … [Read more…]