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Neurogene Doses First Patient With Rett Syndrome Gene Therapy Candidate in High-Dose Cohort

NEW YORK – Neurogene on Tuesday announced that it has treated the first patient with a high-dose of its Rett syndrome gene therapy candidate NGN-401 within a Phase I/II clinical trial and that the patient has so far responded favorably.

Neurogene last year launched the open-label, single-arm Phase I/II clinical trial in which it is enrolling female pediatric patients with Rett syndrome, a rare and progressive disorder affecting brain development and that's caused by mutations in the MECP2 gene. NGN-401 uses an adeno-associated virus serotype 9 vector to deliver a full-length copy of this gene.

New York City-based Neurogene in March said it was planning a second cohort in which patients would receive a higher dose of NGN-401.

The high-dose version of the gene therapy, which the first patient received in May, has been tolerated well, according to the company.

NGN-401 has received orphan drug, fast-track, and rare pediatric disease designations from the US Food and Drug Administration; orphan designation and advanced therapy medicinal product designation from the European Medicines Agency; and innovative licensing and application pathway designation from the UK's Medicines and Healthcare Products Regulatory Agency. The gene therapy candidate was recently selected to participate in the FDA's Support for clinical Trials Advancing Rare disease Therapeutics, or START, pilot program.