Vor(US:VOR)2022-12-16 18:35Vor Bio's Platform and Pipeline - Vor Bio is developing engineered hematopoietic stem cell transplant (eHSC) platform to unlock the potential of targeted therapies with curative intent for hematologic malignancies[73] - The company's lead program, Trem-cel, is a CD33-deleted eHSC designed to protect healthy donor cells from CD33-targeted therapies like Mylotarg and VCAR33[16, 17] - Vor Bio anticipates an IND filing for VCAR33ALLO, an allogeneic CD33-directed CAR-T therapy, in the first half of 2023[15, 45] - The company is exploring additional surface targets such as CD123, EMR2, and CD5, including multiplex genome engineering approaches[15] Trem-cel Clinical Data and Mylotarg - Initial clinical data for Trem-cel + Mylotarg in AML patients showed robust neutrophil engraftment and platelet recovery[28, 29, 30] - In the first patient treated with Trem-cel, CD33 negative cells reached 95% in peripheral blood and 92% in bone marrow by day 28 post-transplant[35] - The first patient achieved complete donor chimerism (100%) in whole blood, myeloid, and NK lineages by day 28[35] - Mylotarg, administered at 0.5 mg/m2 post-HCT, did not show evidence of myelosuppression in the first patient[43] Financials - A recent financing round raised $116 million for the company[73]