After Vor Bio announced initial clinical data from VBP101, its Phase 1/2a multicenter, open-label, first-in-human study of tremtelectogene empogeditemcel, or "trem-cel," in patients with acute myeloid leukemia, or AML, Oppenheimer analyst Matthew Biegler noted that the patient achieved full stem cell engraftment and tolerated subsequent treatment with anti-CD33 antibody-drug conjugate Mylotarg without evidence of its "usual and serious side effects." These data "clear two key trepidations held by investors," namely whether trem-cel is safe to transplant patients with a genetic knockout of the CD33 protein and whether the CD33 knockout provides adequate protection from CD33-targeted therapies, according to Biegler, who thinks VOR33 "could fundamentally upend the field of transplant medicine." He keeps an Outperform rating on Vor shares.
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