Future Research With Stem Cell Therapies in Hematologic Malignancies


The chief executive and chief medical officers of Vor Biopharma discussed the company’s future research and plans.

“Our ability to manipulate the graft occurs ex vivo. So, that puts us primarily in the realm of hematologic malignancies. We're actively looking across the hematologic malignancy spectrum, lymphoid malignancies, acute leukemias, lymphomas, even myelomas all at some point have bone marrow transplant, either autologous or allogeneic stem cell transplant as part of their treatment regimen. So, I think that would be the place to expand immediately outside of myeloid malignancies.”

Vor is developing hematopoietic stem cell (HSC) therapies for the potential treatment of acute myeloid leukemia (AML). A prime example of this is their lead product, VOR33, an allogeneic CRISPR/Cas9 genome-edited HSC engineered to lack the CD33 protein to mitigate off-target effects of anti-CD33 therapies.

The FDA previously granted fast track designation to VOR33 in September 2021.1 The company is currently recruiting for a phase 1/2 trial (NCT04849910) with a target enrollment of 18 participants and primary completion is expected in May 2023.

The company is developing their HSC therapies with the use of their Vor HSC platform. They have also identified additional surface targets with their platform such as CD123 and CLL-1 for future research. The platform is also being used collaboratively with Janssen to improve the tolerability of 1 of their bispecific antibody treatments.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 additional indications the company plans to investigate with their therapies. They also discussed the potential role of their therapies in combination with others.

1. PureTech founded entity Vor Biopharma announces VOR33 granted U.S. Fast Track designation for Acute Myeloid Leukemia. News release. PureTech Health. September 10, 2021. https://www.businesswire.com/news/home/20210909006184/en
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. https://ir.vorbio.com/news-releases/news-release-details/vor-biopharma-announces-collaboration-janssen-develop-engineered
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