Vor Biopharma raises $42M in Series A round to advance AML cell therapy toward clinic

5AM Ventures and RA Capital Management led the round. The company is developing hematopoietic stem cells engineered to be protected from targeted immunotherapies.

A company developing cell therapies in hematology-oncology has raised its first major round of venture capital financing.

Boston-based Vor Biopharma said Thursday that it had raised a $42 million Series A funding round, led by 5AM Ventures and RA Capital Management. Johnson & Johnson Innovation – JJDC, Novartis Institutes for Biomedical Research, Osage University Partners and Vor co-founder PureTech Health participated in the round.

The company said it would use the funding to advance its hematopoietic stem cell-based therapy candidate for treating acute myeloid leukemia toward the clinic and further build its therapy pipeline.

According to the company’s website, it is taking an approach to cell therapy distinguished from traditional CAR-Ts in that it is developing hematopoietic stem cells engineered to be protected from targeted immunotherapies. The cells, it says, are designed to generate healthy, functional cells that are protected from depletion by cancer-targeted therapies. The company said its platform can potentially be used to improve the therapeutic window of cell therapies like CAR-Ts and CAR-NKs, which comprise modified natural killer cells rather than T cells.

“The need for new therapies in hematological malignancies is dire,” Vor co-founder Siddhartha Mukherjee said in a statement. “I am gratified that this discovery from my lab continues to advance towards the clinic. This new platform may enable more patients to benefit from the life-saving potential of targeted immunotherapies.”

Currently, two cell therapies in hematology-oncology have Food and Drug Administration approval: Novartis’s Kymriah (tisagenlecleucel), for acute lymphoblastic leukemia and diffuse large B-cell lymphoma; and Gilead Sciences’ Yescarta (axicabtagene ciloleucel), for DLBCL.

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Whereas ALL, for which Kymriah is approved, is a cancer that mostly affects younger patients, AML is mainly a disease of the elderly. A search on turned up 16 clinical trials of CAR-Ts in AML, mostly in China, with some also in the US. The therapies generally target CD33 or CD123, antigens that are highly expressed in the disease, in contrast to the CD19 antigen that the aforementioned CAR-Ts target in ALL and DLBCL, or the BCMA antigen in multiple myeloma.

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