100% of cancer patients successfully implanted Vor Bio(VOR.US) innovative therapy to enter key trial

Recently, Vor Bio announced positive results from its Phase 1/2 VBP101 trial evaluating the combination of its investigational stem cell therapy tremtelectogene empogeditemcel (trem-cel) with the CD33-targeting antibody drug conjugate (ADC) Mylotarg in patients with relapsed/refractory acute myeloid leukemia (AML). Analysis showed that 100% of patients successfully engrafted trem-cel cells, and patients who received Mylotarg treatment showed an improvement in overall survival. Vor Bio plans to discuss the design of the key trial with the US FDA by the end of this year.

Trem-cel was developed using Vor Bio's proprietary platform, which uses CRISPR/Cas9 gene editing technology to modify hematopoietic stem cells in vitro by knocking out the gene encoding CD33. The hematopoietic stem cells reinfused into patients will produce healthy new cells that do not express the CD33 protein. These healthy cells are like "invisible" cells that will not be mistakenly targeted by the drug. Therefore, higher doses of CD33-targeting therapies can be used to attack tumor cells, while improving specificity and reducing potential side effects.

As of the data cutoff date of July 19, 2024, a total of 18 patients received trem-cel treatment, of whom 10 received Mylotarg treatment. Notably, in the main data of the trial, trem-cel showed reliable engraftment effects, with 100% of patients completing neutrophil engraftment (median time 9 days) and strong platelet recovery (median time 16.5 days). In addition, trem-cel has a high CD33 editing efficiency (median 89%, range 71-94%), and expresses complete myeloid chimerism at day 28.