Gene therapy for rare genetic neurodevelopmental disorders
Total Trials
1
As Lead Sponsor
As Collaborator
0
Total Enrollment
12
NCT07135050
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
Phase: Phase 1/2
Role: Lead Sponsor
Start: Jan 31, 2026
Completion: Jul 31, 2029
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