Neurogene Announces First Pediatric Patients Dosed in US Gene Therapy Clinical Trial

Neurogene Announces First Pediatric Patients Dosed in US Gene Therapy Clinical Trial

Categories: Blog, Clinical Trials, Clinics, Homepage, News, Research

We’re excited to share that today Neurogene announced that two pediatric patients have been dosed in their Phase 1/2 trial of investigational gene therapy NGN-401. For Neurogene’s full press release, click here.

Key Facts:

  • The first two female pediatric patients were dosed sequentially in the 3rd and 4th quarter of this year at Texas Children’s Hospital, an IRSF-designated Center of Excellence.
  • NGN-401 has been well-tolerated with no treatment-emergent procedure-related serious adverse events (SAEs) or transgene-related overexpression toxicity.
  • Pending successful completion of the trial’s upcoming pre-planned independent Data and Safety Monitoring Board review, Neurogene expects to dose a third patient in the first quarter of 2024.
  • Neuorgene is on track to report preliminary clinical data from the first patient cohort in Q4 2024 with data from an expanded number of patients expected in the 2nd half of 2025.

Neurogene’s announcement marks another milestone in what has been an exciting year for Rett syndrome. In 2023 alone, two companies began dosing patients in gene therapy clinical trials and the first-ever drug treatment for Rett syndrome was FDA-approved. We at IRSF promise to continue to push for more research and more treatments until we find solutions for ALL our loved ones living with Rett syndrome.

Thank you to the patients and families who are participating in this clinical trial. You are giving so much hope to our community. Thank you to the scientists and researchers who continue to push exciting treatments and cures forward so that our loved ones can someday do more and struggle less. And thank you to everyone who supports our mission and allows to fund breakthrough research in our pursuit of a world without Rett syndrome.

We are encouraged by these results and remain cautiously optimistic as Neurogene continues its clinical trial. You can learn more about the study and recruiting sites by click here.

IRSF is with you every step of the way. We promise to continue to relentlessly pursue treatments and curative approaches to create a world without Rett syndrome. Questions? Email us at [email protected].