Neurogene Inc. announced on April 2, 2025, the publication of peer-reviewed preclinical data on its proprietary EXACT™ transgene regulation technology in Science Translational Medicine. The findings demonstrate EXACT's ability to precisely regulate gene therapy expression to therapeutic and tolerable levels, including in preclinical models of Rett syndrome.
This publication provides scientific validation for the foundational technology behind NGN-401, Neurogene's lead gene therapy candidate for Rett syndrome. The data support the initiation of the ongoing Phase 1/2 clinical trial for NGN-401, highlighting the technology's potential to overcome limitations of conventional gene therapy in dosage-sensitive neurological disorders.
The EXACT™ platform is designed to deliver therapeutic levels of the MECP2 gene while avoiding overexpression toxicity, a critical challenge in treating Rett syndrome. The peer-reviewed article, titled 'Self-regulating Gene Therapy for Overcoming Gene Dosage Sensitivity in Rett Syndrome,' reinforces the scientific basis for Neurogene's approach.
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