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First-in-human phase 1/2 study of intracerebroventricular AAV9 gene therapy MZ1866 for Pitt-Hopkins syndrome
MOH_2026-03-15_015716Phase 2Recruiting
- Sponsor
- Mahzi Therapeutics
- Start
- Feb 2026
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label trial of MZ-1866 in Pitt-Hopkins syndrome (PTHS) (2 cohorts), sponsored by Mahzi Therapeutics. Primary endpoint: Safety and tolerability of MZ1866. Started Feb 2026; status: recruiting.
02
Drugs
MZ-1866
03
Conditions
Pitt-Hopkins syndrome (PTHS)
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