Bolstering Stem Cells Against Off-Target Effects of Cancer Therapies


The chief executive and chief medical officers of Vor Biopharma discussed their focus on AML, CD33, and their lead program VOR33.

“If the patient relapses, the 2-year survival is less than 20%. We want to radically change that, and make transplant what it should be, which is really a curative therapy for patients such that they can live normal, healthy, productive lives following a bone marrow transplant. We hope that our approach will be able to achieve that.”

Vor Biopharma hopes to develop treatments for acute myeloid leukemia (AML) with their hematopoietic stem cell (HSC) therapies. Vor’s lead product is VOR33, an allogeneic CRISPR/Cas9 genome-edited HSC engineered to lack the CD33 protein to mitigate off-target effects of anti-CD33 therapies. 

VOR33 previously received fast track designation from the FDA earlier in September 2021.1 It is currently being investigated in a recruiting phase 1/2 trial (NCT04849910) with a target enrollment of 18 participants. Primary completion is expected in May 2023.

The company uses their Vor HSC platform to develop their therapies and has identified additional surface targets such as CD123 and CLL-1 for future research. Vor also recently partnered with Janssen to improve the tolerability of 1 of Janssen’s bispecific antibody treatments with the use of the Vor platform.2 

GeneTherapyLive spoke with Robert Ang, MBBS, MBA, chief executive officer, and Christopher Slapak, MD, chief medical officer, both of Vor Biopharma, to learn more about their focus on mitigating toxicities in cancer therapies by protecting HSCs. They also discussed the company’s progress with VOR33.

1. PureTech founded entity Vor Biopharma announces VOR33 granted U.S. Fast Track designation for Acute Myeloid Leukemia. Business Wire. News release. September 10, 2021.
2. Vor Biopharma announces collaboration with Janssen to develop engineered hematopoietic stem cell transplants combined with a bi-specific antibody therapy for acute myeloid leukemia (AML). News release. Vor Biopharma. July 8, 2021.
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