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Neurogene Doses First Two Rett Syndrome Patients in Gene Therapy Trial

NEW YORK – Neurogene has dosed the first two patients in its first-in-human Phase I/II trial of NGN-401, an investigational gene therapy designed to treat female pediatric patients with Rett syndrome, the biotech firm said Thursday.

NGN-401, the firm's lead gene therapy candidate, uses an adeno-associated virus serotype 9 vector to deliver a full-length MECP2 gene. Mutations in this gene cause Rett syndrome, an X-linked, rare, and progressive neurodevelopmental disorder primarily affecting women, many of whom require 24-hour care.

The treatment leverages New York City-based Neurogene's proprietary Expression Attenuation via Construct Tuning (EXACT) gene regulation technology, which aims to deliver a desired level of transgene expression and limit transgene-related toxicities.

"We are encouraged by the tolerability profile observed in our first two pediatric patients and look forward to collecting sufficient follow-up data on a larger number of patients to inform the therapeutic potential of NGN-401," Neurogene Founder and CEO Rachel McMinn said in a statement.

In the open-label, single-arm Phase I/II trial, Neurogene will assess the safety and tolerability of NGN-401 in female Rett syndrome patients aged 4 to 10 years old. The first two patients received NGN-401 recently, and Neurogene expects to dose a third patient in Q1 2024.

Neurogene plans to enroll a total of five patients in the first cohort of the trial and expand enrollment based on analysis of additional data and review by health authorities. The firm said it will report preliminary data from the first cohort of patients by the end of 2024.