NGN-401 is given to paediatric patients using Neurogene’s EXACT gene regulation technology. Credit: Charlein Gracia / Unsplash.
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The US Food and Drug Administration (FDA) has granted clearance for Neurogene’s investigational new drug (IND) application for NGN-401 to treat Rett syndrome.

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

Developed with the University of Edinburgh, the EXACT self-contained technology enables MeCP2 protein therapeutic levels while avoiding overexpression-related toxicities.

The transgene regulation technology can be tuned to provide a desired transgene expression level in a narrow range.

NGN-401 showed robust therapeutic and safety benefits while delivering MECP2 to important regions of the brain that are affected by Rett syndrome in a comprehensive preclinical programme.

Neurogene stated that the FDA IND clearance allows it to commence a Phase II/II trial of NGN-401 in female paediatric Rett syndrome patients.

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The multi-centre, open-label, single-arm clinical trial has been designed to evaluate the efficacy, tolerability, and safety of a single dose of NGN-401 delivered using a one-time ICV procedure.

Neurogene CEO and founder Rachel McMinn said: “Rett syndrome is a particularly challenging disorder for gene therapy because of the requirement to deliver therapeutic levels of MECP2 without also triggering significant side effects associated with too much gene expression.

“We believe the preclinical profile for NGN-401 is highly compelling, with the strongest results generated to date across multiple animal models.

“FDA clearance of NGN-401 represents a significant milestone for Neurogene and the Rett syndrome community and underscores our commitment to turn devastating neurological diseases into treatable conditions and to improve the lives of patients and families impacted by these rare diseases.”

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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