Dec. 1st 2022–

The California Institute for Regenerative Medicine (CIRM.gov) has awarded a $4 million dollar grant to Mahzi Therapeutics (mahzi.com) to accelerate their work in Pitt Hopkins Gene Therapy.

Mahzi, a rare disease therapeutic company started by esteemed scientists from the successful biotech, Ultragenyx, launched earlier this year with Pitt Hopkins as one of their first 3 pipeline indications. We were honored they chose us, and are even more honored and grateful that they saw so much promise in the work of Drs. Alysson Muotri and Fabio Papes (PTHS Uncle) at UCSD that they applied for this grant. For the CIRM to also see the promise and make this unprecedented investment into Pitt Hopkins translational work is absolutely transformative for our community! We are hopeful to see this therapy launch in human clinical trials in 2024.

Read more about the grant below:
https://www.cirm.ca.gov/our-progress/awards/development-gene-therapy-treatment-pitt-hopkins-syndrome-phs-translating-animal?fbclid=IwAR3sTU-OJd69lYIvB34Pz8EaYUKgLCjLRUSjRCcw5rPkLN0xdWKuL6ET6Vc