Mahzi Therapeutics has dosed the first patient in its Phase I/II UNITE study assessing MZ-1866, an investigational gene therapy targeting Pitt Hopkins syndrome.
The multicentre, open-label, global trial will investigate the tolerability, early efficacy, and safety of a single MZ-1866 administration in patients with genetically confirmed Pitt Hopkins syndrome.
It aims to enrol around 12 participants at five sites across Spain, Israel, and the US.
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They will receive a single dose of the therapy delivered through intracerebroventricular administration.
Alongside primary safety evaluation, the study will assess exploratory endpoints, including cognitive, communication, developmental, and motor function outcomes.
Pitt Hopkins syndrome is a rare condition that affects roughly one in 34,000 to 41,000 individuals.
Mahzi Therapeutics reported that an estimated 8,000 people in the US are living with this condition caused by mutations in the transcription factor 4 (TCF4) gene.
Symptoms may include autism, developmental delays, hypotonia, ataxia, breathing irregularities such as apnoea or hyperventilation, severe gastrointestinal complications, and occasionally epilepsy.
MZ-1866 is an adeno-associated virus serotype 9 (AAV9) TCF4 therapy, constructed by the insertion of TCF4 isoform B into an AAV9 expression cassette under the regulation of multimer E box sequences.
Mahzi Therapeutics CEO Yael Weiss said: “Dosing the first patient is a significant achievement for the MZ-1866 programme and for Mahzi’s evolution as a clinical-stage company. We look forward to advancing this programme to address a significant unmet medical need for patients with Pitt Hopkins syndrome and their families.”
MZ-1866 was developed in partnership with the Muotri Lab at the University of California, San Diego.
The Phase I/II study received funding from the California Institute for Regenerative Medicine (CIRM).
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