Neurogene press release: Rapid enrolment and expansion

Neurogene has published a press release today with an update to their Rett syndrome gene therapy clinical trial of NGN-401. This update is mainly focused on their plans to expand the ongoing children’s trial including that they have been given authorisation to enrol patients more quickly.

You can read the full press release here.

You can read the community letter here.

Key takeaways

• 3 children with Rett syndrome have been dosed so far in the USA
• There have been no adverse safety issues either from the gene therapy itself or from the procedure
• Another 5 patients will receive the same dose to make up Cohort 1
• These patients can now be dosed without a delay between patients

In addition, another group or ‘cohort’ has now been added to the trial protocol:

• This group (Cohort 2) will receive a higher dose of the gene therapy
• The first patient is expected to be dosed in Q2 2024 (April-June)
• The first three patients in Cohort 2 will be staggered meaning that they will be dosed with a delay between patients.
• After the first three patients have been dosed, the Data and Safety Monitoring Board will review
• All being well they will then move on to dose the rest of the patients without the delay between patients
• A preventative immunosuppression regimen has been added for Cohort 2
• Cohort 1 immunosuppression remains unchanged with corticosteroids alone

As mentioned in the Community letter, these are planned changes to the protocol. A similar protocol amendment was also submitted to the UK regulator, the MHRA.

• The MHRA has already approved a UK clinical trial of NGN-401 which will enable eligible UK patients to take part in the trial
• The next steps are to establish the first clinical trial site in the UK, which may take several months
• UK patients would be included in the number of patients in the trial globally (16 participants)
• Further communications will be shared at the time the clinical trial site is open