Vor Bio reports initial clinical proof-of-concept for trem-cel; first AML patient engrafts normally

Vor Biopharma Inc.

First patient received trem-cel dose of 7.6x10^6 CD34+ cells/kg with 88% CD33 editing efficiency.
Neutrophil engraftment at 10 days, platelet recovery Day 22; >90% of PBMCs CD33-negative, 100% donor chimerism.
No infusion reactions or treatment-related adverse events observed post-transplant.
Mylotarg 0.5 mg/m^2 tolerated with no neutropenia or liver enzyme changes through Day 20.
Trial continues enrolling; additional data expected in 2023; conference call Dec 7 at 8am ET.

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