Neurogene has announced encouraging interim clinical data in the paediatric cohort from its ongoing Phase I/II trial of NGN-401 gene therapy for females with Rett syndrome.
The study included subjects aged between four and ten years, with data indicating improvements in developmental milestones across all participants, irrespective of their initial disease severity.
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The safety data from ten subjects indicated that NGN-401 at a dose of 1E15 vg was generally well tolerated, exhibiting a favourable safety profile across both the paediatric arm and the adolescent/adult group aged 11 years and older.
All adverse events related to the treatment were found to be moderate or mild in severity, and most represent known potential risks associated with adeno-associated viruses (AAV), and they have either resolved or are in the process of resolving.
The company also reported no haemophagocytic lymphohistiocytosis (HLH) evidence in any of the subjects.
Neurogene founder and CEO Rachel McMinn said: “Updated interim data from our Phase I/II trial of NGN-401 show that all paediatric participants, regardless of baseline disease severity, have gained developmental milestones/skills or experienced functional gains, with durability and continued skill acquisition over time.
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By GlobalData“These skill gains far exceed the bar for our Embolden registrational trial, and coupled with NGN-401’s tolerability profile at the 1E15 vg dose, create the potential for a best-in-class gene therapy for Rett syndrome.
“It is gratifying to see the benefits of our strategy to use local delivery to the brain coupled with the naturally occurring full-length human gene as a key to unlocking meaningful efficacy in this devastating disorder.”
The efficacy data from eight subjects in the paediatric cohort indicated that all individuals experienced improvements across the full range of disease severity.
In total, there was an accumulation of 35 developmental milestones or skills achieved across key clinical areas of Rett syndrome, including ambulation and gross motor skills, language and communication, as well as hand function and fine motor skills.
Previously announced enhancements in the Clinical Global Impression-Improvement have remained stable. Further clinical data will be released in 2026.
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