MZ-1866

MZ-1866 is an investigational adeno-associated virus serotype 9 (AAV9) gene replacement therapy designed to treat Pitt Hopkins syndrome, a rare neurogenetic disorder caused by haploinsufficiency or mutations in the transcription factor 4 (TCF4) gene. Developed by Mahzi Therapeutics in collaboration with the Muotri Lab at the University of California, San Diego (UCSD), MZ-1866 delivers a functional copy of the TCF4 isoform B gene under the regulation of multimer E-box sequences. The therapy is administered as a single dose via intracerebroventricular (ICV) injection to directly target the central nervous system, aiming to restore TCF4 expression and improve cognitive, communication, and motor functions. It is currently being evaluated in the Phase 1/2 UNITE clinical trial.

Other names

AAV9-based gene therapy for TCF4 deficiencyAAV-9-based gene therapy for TCF4 deficiencyAAV 9-based gene therapy for TCF4 deficiencyAAV9-TCF4 gene therapyAAV-9-TCF4 gene therapyAAV 9-TCF4 gene therapyAAV9-TCF4 replacement therapyAAV-9-TCF4 replacement therapyAAV 9-TCF4 replacement therapy