Mahzi doses the primary affected person within the MZ-1866 trial for Pitt Hopkins syndrome
Mahzi Therapeutics has dosed the primary affected person in its Part I/II UNITE trial evaluating MZ-1866, an investigational gene remedy concentrating on Pitt Hopkins syndrome.
The multicenter, open-label, international examine will examine the tolerability, early efficacy and security of a single administration of MZ-1866 in sufferers with genetically confirmed Pitt Hopkins syndrome.
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Extra data
The aim is to enroll roughly 12 individuals in 5 places in Spain, Israel and the US.
They obtain a single dose of the remedy by way of intracerebroventricular administration.
Along with the first security analysis, the examine will assess exploratory endpoints together with cognitive, communication, developmental and motor perform outcomes.
Pitt Hopkins syndrome is a uncommon situation that impacts roughly one in 34,000 to 41,000 folks.
Mahzi Therapeutics reported that an estimated 8,000 folks within the US reside with the situation, brought on by mutations within the transcription issue 4 (TCF4) gene.
Signs might embrace autism, developmental delays, hypotonia, ataxia, irregular respiratory reminiscent of apnea or hyperventilation, extreme gastrointestinal issues and typically epilepsy.
MZ-1866 is an adeno-associated virus serotype 9 (AAV9) TCF4 remedy constructed by the insertion of TCF4 isoform B into an AAV9 expression cassette below the regulation of multimeric E-box sequences.
Yael Weiss, CEO of Mahzi Therapeutics, mentioned, “Dosing the primary affected person is a big achievement for the MZ-1866 program and for Mahzi’s evolution as a clinical-stage firm. We look ahead to advancing this program to handle a big unmet medical want for sufferers with Pitt Hopkins syndrome and their households.”
MZ-1866 was developed in collaboration with the Muotri Lab on the College of California, San Diego.
The Part I/II examine was funded by the California Institute for Regenerative Drugs (CIRM).
