Neurogene update

It is with deep sadness that we share that a child on the Neurogene Rett syndrome gene therapy clinical trial has died.

This child was the third patient to be dosed with Neurogene’s high dose gene therapy. The other two patients previously dosed have experienced no adverse reactions.

More information can be found in Neurogene’s letter to the patient community here.

The thoughts of Rett families across the world will be with this family as they grieve for their precious daughter.

Families will also be understandably concerned about what this means for the Neurogene gene therapy program moving forward.

Neurogene has underscored their ongoing commitment to delivering meaningful treatment for patients affected by Rett syndrome.

The FDA has approved the continuation of the NGN-401 program at the low dose level. For clarity, five patients have now been dose with low dose NGN-401 and three at the high dose, including the little girl who has died.

The rare and life-threatening  immune reaction to AAV (the virus which carries the gene therapy into the body) has not been seen at the low dose level at which the trial will continue.