What is the UNITE study investigating?

The UNITE study is investigating MZ-1866, an investigational gene therapy for Pitt Hopkins syndrome, assessing its tolerability, early efficacy, and safety.

How rare is Pitt Hopkins syndrome?

Pitt Hopkins syndrome is a rare condition, affecting roughly one in 34,000 to 41,000 individuals, with an estimated 8,000 people living with it in the US.

Who developed the MZ-1866 gene therapy?

The MZ-1866 gene therapy was developed by Mahzi Therapeutics in partnership with the Muotri Lab at the University of California, San Diego.

Home / Health / Gene Therapy Trial for Rare Pitt Hopkins Syndrome Begins

Gene Therapy Trial for Rare Pitt Hopkins Syndrome Begins

26 Feb

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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.

Gene Therapy Trial for Rare Pitt Hopkins Syndrome Begins

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.