Neurogene Announces First Patient Dosed in High-Dose Cohort of NGN-401 Gene Therapy Clinical Trial for Rett Syndrome

On June 18th, Neurogene provided an update on the first “high dose” patient in their NGN-401 trial, as well as the first 3 patients in their low dose cohort.

You can read Neurogene’s press release here

Here are our key takeaways

High dose cohort:
The first patient in the high dose trial has been dosed.
The high dose has been well tolerated, and there have been no new treatment-related adverse events since dosing in May.

Low dose cohort:
NGN-401 continues to have a favorable safety profile.
There have been no new treatment-related adverse events.

When will we next know more?
Neurogene says they are on track to release efficacy data for the low-dose cohort of patients in the last quarter of 2024.