It feels almost surreal to be able to share what is without a doubt the most remarkable milestone in the history of the Pitt Hopkins research. Today we are announcing that the first patient has been dosed with Mahzi’s gene-replacement therapy, MZ-1866, and is doing well. Eight years in the making, this extraordinary achievement reflects countless hours of dedication and an unwavering belief in hope.

This win belongs to so many, and our gratitude for all of them is beyond words.

Our first patient and their family are quiet heroes. Their courage and determination to pursue a better life for their child will help open the door to a better future for so many others.

The Muotri Lab, led by Dr. Alysson Muotri at UCSD, whose visionary research not only demonstrated that gene therapy could work in cellular models, but also reminded us that hope was real.

Yael Weiss and her devoted team at Mahzi Therapeutics, who took this drug out of academia and made it a top-tier product ready for human trials.

Our incredible donors who have stuck with us through thick and thin.

The Pitt Hopkins community, who believed in us even when the road to treatment felt impossibly long.

And above all, our Pitt Hopkins children—you are the true heroes. Your remarkable courage, resilience, and heart inspire us every day to keep moving forward, not only to hope for miracles, but to fight for them. Always.

Read the Full Press Release here.

With Sincere Gratitude,

Pitt Hopkins Research Foundation