Mahzi Therapeutics Gene Therapy Trial

Licensed from PHRF funded work at UCSD’s Muotri Lab

Gene therapy is no longer a dream—it’s becoming a reality. Building on foundational research from the Muotri Lab at UC San Diego, Mahzi Therapeutics is preparing to launch the first-ever gene therapy trial for Pitt Hopkins Syndrome. Recruitment is anticipated to begin in late 2025 or early 2026. The trial will include two age cohorts—ages 2–11, and 12–25—with the oldest group enrolling first. Trial site selection is underway, with both U.S. and international locations under consideration. This is a historic moment for our community, and we’ll be sharing more details as they unfold.

Read more about Mahzi here.