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Muotri Lab brain organoids laid the foundation for Pitt Hopkins syndrome gene therapy trial
31 January 2026 Mahzi Therapeutics-sponsored Phase 1/2 study to assess safety and efficacy of MZ-1866 gene therapy for children and adults with Pitt Hopkins syndrome is about to start. The trial was announced by Mahzi in 2025, highlighting the sponsor's collaboration with the Pitt Hopkins Research Foundation and the Muotri Lab at UC San Diego in developing this long awaited treatment. Pitt-Hopkins syndrome (PHS) is a rare genetic disorder caused by mutations in the tran
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