Neurogene Releases Update on NGN-401 Clinical Trial High Dose

I’m writing to inform our community about an update that Neurogene gave this morning on its NGN-401 gene therapy clinical trial. You can read more details in Neurogene’s release in the link below.

The key information that Neurogene shared is that one patient, the child who was the third to receive high-dose gene therapy, experienced an immune response to the AAV.  She is alive but is in critical condition. The high-dose arm of the trial is being discontinued. The FDA has allowed the company to continue the low dose. The adult trial will also enroll low dose.

This program got its start at RSRT over ten years ago in our Gene Therapy Consortium. Alongside all of our community, we applauded as the trial got started and waited anxiously for news.  The first update of efficacy came last week and we were buoyed by the signs of improvement. The news that a child in the trial suffered a reaction to the high dose AAV fills us with sadness. We join the entire Rett community in sending our most heartfelt best wishes to the child and her family for a speedy and complete recovery. We are profoundly grateful to the brave children and their families who participate in clinical trials.

This situation is a sobering reminder that clinical trials, while they carry the potential for great positive change for all our loved ones, also come with risks. Our work continues, and we remain committed to advancing genetic medicines for Rett syndrome.