New Collaboration to Assess Stem Cell Therapy With Bispecific Antibody in AML


Janssen hopes that Vor’s stem cell therapy will allow their bispecific antibody to be better-tolerated.

Janssen has partnered with Vor Biopharma to improve the tolerability of a bispecific antibody treatment with one of Vor’s stem cell therapies.1

Vor Biopharma will investigate the effect of its “invisible” engineered hematopoietic stem cell (eHSC) transplant platform on the tolerability of a bispecific antibody in development for acute myeloid leukemia (AML) by Janssen.

“We are thrilled to enter into this collaboration with Janssen as we continue to explore our platform’s potential to pair with a broad spectrum of targeted therapy modalities for the treatment of patients with blood cancer,” said Tirtha Chakraborty, PhD, chief scientific officer, Vor Biopharma, in a statement. “We believe this unique combination will leverage each technology’s strengths, while protecting patients against off-target effects of these powerful immunotherapies.”

Janssen did not disclose which bispecific antibody treatment would be studied, although their pipeline currently includes a CD33xCD3 bispecific antibody in phase 1 development in AML and myelodysplastic syndrome (MDS) called JNJ-67571244.

Vor Biopharma’s eHSC therapies, including their lead asset VOR33, aim to create next-generation, treatment-resistant transplants that will aid the use of targeted therapies. The eHSCs are designed to be treatment-resistant to complementary targeted therapies so that therapies will spare healthy cells while affecting target cells. The therapies are created by taking healthy matched donor stem cells, deleting the gene that encodes for the therapy’s target, and delivering the eHSCs to the patient to be engrafted into bone marrow. Blood cells will then be produced that lack the surface receptor targeted by the therapy.

READ MORE: Novel T-Cell Therapy MT-401 Under Evaluation in Post-Transplant AML

Vor is currently investigating using VOR33 with both its own CD33-directed chimeric antigen receptor T-cell (CAR-T) therapy as well as Pfizer’s Mylotarg. A planned phase 1/2 study of VOR33 and Mylotarg is set to begin soon and evaluate the combination of treatments in patients with AML who are at high risk for leukemia relapse following HSC transplantation.

In June 2021, Vor Biopharma announced their plans to build an in-house clinical manufacturing facility in Cambridge, Massachusetts to support the development of their stem cell therapies as well as their CAR- T therapeutic candidates for patients with hematological malignancies.2 They anticipate that the facility will be operational in 2022. 

“Vor’s novel approach to cancer treatment demands an equally novel approach to cell therapy manufacturing. We are thrilled to construct our own manufacturing facility in our existing location, which will give us end-to-end oversight over drug product for our planned clinical trials and nicely complement our global supply chain,” said Sadik Kassim, PhD, chief technology officer, Vor Biopharma, in a statement at that time.2 “With this new facility, our manufacturing teams will be seamlessly integrated within our wider organization, a crucial component of our strategy as we begin treating patients living with devastating blood cancers.”

1. 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.
2. Vor Biopharma to build state-of-the-art cell therapy drug production site in Cambridge MA. News release. Vor Biopharma. June 17, 2021.
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