Neurogene announced the following updates today to their pediatric gene therapy trial of NGN-401.
- Three patients have been dosed to date in the low dose cohort and NGN-401 has been well tolerated
- The size of the trial has been expanded from 5 patients to 16 with 8 patients expected to be enrolled in the low dose cohort and 8 patients in the high dose cohort
- Age range is 4 to 10 years old
- Patients will be recruited from the US and UK
- The remaining 5 patients in the low dose cohort can now be dosed concurrently with no waiting period between patients
- The first 3 patients in the high dose cohort will be dosed in a staggered manner with the first patient expected to be dosed in 2nd quarter of this year
We encourage you to read Neurogene’s community letter and press release below for additional details.