J&J, Vor ally to assess bispecific and stem cell therapy in AML

Johnson & Johnson has teamed up with Vor Biopharma to study one of its bispecific antibodies in combination with technology designed to improve tolerability. 

Vor, which was founded by Siddhartha Mukherjee, is built on a way to make healthy cells invisible to targeted treatments. The process entails taking stem cells from a healthy matched donor, deleting the gene that encodes for the target of the therapy and giving the engineered cells to the patient. When the engineered cells engraft into bone marrow, the patient will make blood cells that lack the surface receptor targeted by the therapy, thereby preventing on-target toxicity.

J&J sees the technology as a good fit for a bispecific antibody it is developing in the treatment of acute myeloid leukemia (AML). The statement lacks further details of the J&J drug. J&J’s pipeline includes a CD33xCD3 bispecific antibody that is in phase 1 development in AML and myelodysplastic syndrome.

That drug, JNJ-67571244, would be a natural fit for Vor’s lead asset VOR33. As the name suggests, VOR33 is designed to spare patients from on-target toxicity when treated with anti-CD33 therapies. Vor is exploring the effects of using VOR33 with Pfizer’s Mylotarg and its own CD33-directed CAR-T, but the cells could be equally applicable to JNJ-67571244.

J&J has secured the chance to assess the combination of its bispecific and Vor’s technology without risking much. The statement to disclose the deal makes no mention of any financial terms. J&J and Vor will retain all rights to their respective programs and platforms.  

Vor will work on the J&J project in parallel to its in-house programs. A phase 1/2 study of VOR33 and Mylotarg is set to start soon, giving Vor an early look at the effects of its candidate in AML patients who are at high-risk for leukemia relapse following hematopoietic cell transplantation. Vor raised $203 million in an IPO early this year to fund the work.