Skip to main content

Neurogene Shares Key Information on Gene Therapy Clinical Trial with the Rett Community

April 18, 2023

In January Neurogene announced that the FDA had approved its Investigational New Drug (IND) application for a gene therapy clinical trial for Rett syndrome. It was a moment we had been working toward and looking forward to for a long time at RSRT. Now, we are pleased to share a letter to the Rett community from Neurogene, published today, providing important new information on the clinical trial. Key highlights from the letter include:

  • The age range for participants in the trial will be 4- to 10-years old.
  • The initial phase of the clinical trial will enroll five participants. Neurogene anticipates enrolling more participants as the trial progresses.
  • Enrollment is set to begin in summer or fall of this year.
  • The trial will be limited to participants living in the US. Neurogene is exploring the opportunity for additional clinical trial sites outside the US.
  • Currently, the trial will not be able to enroll participants who are taking, or have ever taken, trofinetide.

Please read the entire letter from Neurogene here.

Just a few years ago a clinical trial in gene therapy was something we could only have dreamed of for Rett syndrome. The fact that it is happening and that the details of the trial are being fine-tuned is indicative of the remarkable progress that’s being made with potential genetic medicines for Rett. We are, as always, deeply grateful to our donors and to the families that fundraise for RSRT for helping to make this progress possible. We look forward to sharing additional updates on the clinical trial in the months ahead.