Gene therapy breakthrough? first human trial launches for rare brain disorder
Disease control
Recruiting now
This study tests a new gene therapy called MZ-1866 for children and adults with Pitt Hopkins Syndrome, a rare genetic condition that affects development and breathing. The therapy is given as a single injection into the fluid around the brain. The main goal is to see if it is saf…
Phase: PHASE1, PHASE2 • Sponsor: Mahzi Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:31 UTC