Neurogene doses first paediatric patients in US clinical trial of NGN-401 gene therapy 


November 30th 2023-Neurogene has announced today that they have dosed the first two female patients with Rett syndrome in their US clinical trial of their gene therapy product NGN-401 for children with Rett syndrome ages 4-10.

To date, NGN-401 has been well-tolerated with no significant adverse events or signs of over-expression. This is a first in human study and the focus of the clinical trial is primarily safety.

The first two patients were dosed sequentially in the third and fourth quarter of 2023 at Texas Children’s Hospital under Primary Investigator, Dr Daniel Curry MD. The first cohort expected to enroll a total of five female children with planned expansion pending additional data.

Neurogene Founder and CEO, Rachel McMinn, Ph.D., said, ‘We are encouraged by the tolerability profile observed in our first two paediatric patients and look forward to collecting sufficient follow up data on a larger number of patients to inform the therapeutic potential of NGN 401 which we believe could serve as best in class therapy. We remain on track to report preliminary clinical data from the first cohort of patients inthis trial in the fourth quarter of 2024 with additional data for an expanded number of patients expected in the second half of 2025.’

Dr McMinn added,’ “On behalf of the entire Neurogene team, we extend our gratitude to all those making this study possible, with special appreciation for the patients and their families. Your resilience, courage, and support not only contribute to the progress of this research, but also inspire hope within the entire Rett syndrome community. Together, we have the potential to make a meaningful impact on improving the many lives affected by this devastating disease.”

Read the full press release here:

Key take aways from today’s announcement:

  1. NGN-401 has now been given to two US patients with Rett syndrome between the ages of 4-10.
  2. There have been no adverse reactions to date.
  3. No information regarding efficacy/whether there have yet been any improvements in the patients’ condition has been shared.
  4. At this time there is no information available about trial sites outside the USA.
  5. The company plan to dose 5 patients initially and then expand the trial cohort as appropriate once data is independently reviewed.

Did you know?

Through our longstanding partnership with RSRT, Reverse Rett has provided a significant level of funding for Professor Stuart Cobb, now the Chief Scientific Officer of Neurogene, whose work led to the Neurogene NGN 401 program.  

The Cobb Lab alone has received over £1.4million from Reverse Rett over the last decade enabling this work to move from the laboratory bench to human clinical trials

We are delighted for Stuart and send our congratulations to him, all his colleagues at the Cobb Lab at the University of Edinburgh and all the team at Neurogene for all their efforts to bring these potentially life-changing treatments to the international Rett community.