According to the smart finance app, Vor Bio recently announced the positive results of the Phase 1/2 VBP101 trial, which investigates the combination of tremtelectogene empogeditemcel (trem-cel) and the CD33-targeted antibody-drug conjugate (ADC) Mylotarg for the treatment of relapsed/refractory acute myeloid leukemia (AML) patients. The analysis showed that 100% of patients successfully received trem-cel cell transplantation and exhibited improved relapse-free survival after receiving Mylotarg treatment. Vor Bio plans to discuss the design of the relevant key trials with the US FDA by the end of the year.

Trem-cel is developed based on Vor Bio's proprietary platform. This platform utilizes CRISPR/Cas9 gene editing technology to modify hematopoietic stem cells in vitro by deleting the CD33 gene. The hematopoietic stem cells that are reintroduced into the patient's body will generate healthy cells that do not express the CD33 protein. These healthy cells are like wearing an "invisibility cloak" and will not be mistakenly targeted by drugs. Therefore, higher doses of CD33-targeted therapy can be used to combat tumor cells, increasing specificity while reducing potential side effects.

As of the data cutoff date of July 19, 2024, a total of 18 patients have received trem-cel treatment, of which 10 patients have also received Mylotarg treatment. Of note, in the primary data of the trial, Trem-cel demonstrated reliable engraftment, with 100% of patients achieving neutrophil engraftment (median time of 9 days) and showing robust platelet recovery (median time of 16.5 days). In addition, trem-cel showed a high CD33 editing efficiency (median 89%, range 71-94%) and expressed complete myeloid chimerism by day 28.