Vor Biopharma (VOR) Investor Presentation - Slideshow

Company Vision and Platform - Vor Biopharma's vision is to cure blood cancer through transformative cell engineering, utilizing a VOR platform that combines Hematopoietic Stem Cells (HSCs), genome engineering, and CAR-T cell therapies[6] - The company is developing engineered HSCs (eHSCs) as a solution to remove target expression on healthy cells, making cancer killing more cancer-specific[7, 8] - Vor's technology-driven platform vision includes VOR33 + VCAR33, multiplexed HSCs + dual-specific CAR-T, exploring targets beyond CD33, and exploring next-generation technologies like genome engineering and companion therapeutics[49] Pipeline and Clinical Development - The company's pipeline includes VOR33 (eHSC targeting CD33) for AML, MDS, and MPN, and VCAR33 (CAR-T targeting CD33) as a bridge-to-transplant for AML[13] - VOR33 is expected to have initial clinical data in the first half of 2022, and a development candidate selection for MDS and MPN[13] - The VOR33/VCAR33 treatment system is planned for an IND filing in the second half of 2022, following initial VOR33 and NMDP clinical data[13] VOR33 and CD33 Target - Preclinical validation of CD33 deletion in HSCs shows successful homing, engraftment, survival, and resistance to toxic therapy[18] - VOR33 shows no observed impact on cell populations or function, including B cells, myeloid cells, and phagocytosis[21, 23, 25] - In vitro studies show VOR33 provides resistance to CD33 therapy, with approximately 70-fold resistance to Mylotarg[33] Manufacturing and Clinical Trial - Vor is building a fully integrated manufacturing facility for eHSC and CAR-T drug products, with a streamlined cell manufacturing process for VOR33 that takes approximately 3 days, with a 7-10 day vein-to-vein time[35, 37] - VBP101 is a VOR33+Mylotarg Phase 1/2a clinical trial for transplant-eligible AML patients in remission but at high risk of relapse[38, 39]