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.

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.

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