A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome

Inclusion Criteria:

• Diagnosis of typical Rett syndrome with a documented disease-causing mutation in the methyl-CpG-binding protein 2 (MECP2) gene
• Current anti-epileptic drug regimen has been stable for at least 12 weeks
• Participant and caregiver must reside within a 2-hour drive of the study center for at least 3 months following treatment

Exclusion Criteria:

• Normal or near normal hand function
• Has a current clinically significant condition other than Rett syndrome
• Presence of a concomitant medical condition that precludes intracerebroventricular administration, or use of anesthetics needed for study related procedures
• Grossly abnormal psychomotor development in the first 6 months of life
• A history of other genetic disorders or neurological conditions, such as stroke, brain tumor, or autoimmune processes affecting the central nervous system

Other inclusion or exclusion criteria apply.