| Therapeutic Translational Research Projects | Dr. Allyson Berent | Development of a Gene Therapy for the Treatment of Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting |
$3,800,760 |
| Clinical Trial Stage Projects | Dr. Yael Weiss | Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, a gene therapy for Pitt Hopkins Syndrome |
$8,000,000 |
| Late Stage Preclinical Projects | Dr. Yael Weiss | Development of a Gene Therapy for the Treatment of WWOX related epileptic encephalopathy (WOREE) |
$4,000,000 |
