​Dear GP2C and Rett University Community, some important news from our friends at the Rett Syndrome Research Trust regarding Neurogene clinical trials… PROGRESS!

https://reverserett.org/news/articles/neurogene-key-information-april-2023/?mc_cid=dbcff49a36&mc_eid=da8ecbfc1f

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

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.

 

Regards, Kevin