Home / Health / Gene Therapy Trial for Rare Pitt Hopkins Syndrome Begins
Gene Therapy Trial for Rare Pitt Hopkins Syndrome Begins
26 Feb
Summary
- First patient dosed in Phase I/II UNITE study for Pitt Hopkins syndrome.
- Study assesses tolerability and early efficacy of novel gene therapy.
- Rare condition affects approximately 1 in 34,000 individuals.
Mahzi Therapeutics has dosed the first patient in its Phase I/II UNITE study, evaluating MZ-1866, an investigational gene therapy for Pitt Hopkins syndrome. This multicenter, open-label trial is being conducted globally, enrolling approximately 12 participants across Spain, Israel, and the US.
The study aims to assess the tolerability, early efficacy, and safety of a single administration of MZ-1866, delivered via intracerebroventricular means. Exploratory endpoints will also examine cognitive, communication, developmental, and motor function outcomes.
Pitt Hopkins syndrome is a rare genetic disorder affecting approximately 8,000 people in the US, stemming from mutations in the TCF4 gene. Symptoms can include autism, developmental delays, hypotonia, ataxia, breathing irregularities, and epilepsy.
Developed in partnership with the Muotri Lab at the University of California, San Diego, and funded by the California Institute for Regenerative Medicine (CIRM), MZ-1866 represents a significant step in addressing a critical unmet medical need for affected families.
