Vor Bio reports positive trem-cel + Mylotarg data; plans pivotal trial

Vor Biopharma Inc.

Phase 1/2 VBP101: 18 patients received trem-cel; 10 received Mylotarg as of July 19, 2024 cut-off.
100% engraftment (median 9 days); CD33 editing efficiency median 89%; full myeloid chimerism at Day 28.
Shielding maintained neutrophil/platelet counts across Mylotarg doses 0.5, 1, 2 mg/m^2.
Early relapse-free survival benefit vs high-risk AML comparators; only 4 relapses recorded.
New preclinical ADC VADC45 targeting CD45; IND-enabling studies planned; FDA pivotal trial discussion by year-end.

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