Christopher Slapak, MD

“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.

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.

 spoke with Robert Ang, MBBS, MBA, chief executive officer, and Christopher Slapak, MD, chief medical officer, both of 

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

Future Research With Stem Cell Therapies in Hematologic Malignancies 

“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. 

 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 

 to improve the tolerability of 1 of Janssen’s bispecific antibody treatments with the use of the Vor platform.

 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. 

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

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

Christopher Slapak, MD

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