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Cambridge biotech raises $110 million to advance treatment for blood cancer

Vor Biopharma CEO Robert Ang.Vor Biopharma

COVID-19 has upended the economy, but biotechs with promising medical treatments are still attracting venture capital.

Take Vor Biopharma. The Cambridge startup cofounded by Dr. Siddhartha Mukherjee, the oncologist and Pulitzer Prize-winning author, said Tuesday it has raised $110 million in new funding that it hopes to use to start clinical trials of a new approach to treating acute myeloid leukemia, or AML, a cancer of the blood and bone marrow. The company raised $42 million in February of last year.

“The pandemic certainly provided a set of challenges, and it’s unusual to raise money when you cannot shake hands with investors . . . before parting with their hard-earned funds,” said Dr. Robert Ang, Vor’s chief executive. That Vor raised as much as it did, he said, “is testament to the novelty of our science and the quality of the company.”


Current approaches to treating AML include a stem cell transplant in which cancerous bone marrow is replaced with highly specialized cells, called hematopoietic stem cells, that develop into healthy bone marrow. Doctors then use a variety of drugs, including immunotherapy, to attack remaining cancer cells or keep them at bay by targeting a signature protein that they express.

The problem is that some healthy cells also make this protein, known as CD33, and can be ambushed by the medicines. Vor has figured out a way to delete that protein from healthy cells through gene editing, so that only cancerous cells are killed.

The biotech is named Vor for the Norse goddess of wisdom, who was known as “the careful one,” according to the startup’s website. The company’s experimental method for treating cancer is meant to take care of healthy cells.

“Vor has an elegant approach to engineering hematopoietic stem cells that we believe is amongst the most promising innovations in oncology,” said Dr. Joshua Resnick, managing director of RA Capital Management, which led the latest fund-raising round.


New investors in the startup include Fidelity Management & Research Company, Pagliuca Family Office, and Alexandria Venture Investments. Previous investors who ponied up again include 5AM Ventures, Johnson & Johnson Innovation, Osage University Partners, and cofounder PureTech Health.

Vor’s approach to fighting blood cancers was the subject of a “proof of concept” study in May 2019 in the journal Proceedings of the National Academy of Sciences. The article was co-written by Mukherjee, Vor’s scientific founder. He is an assistant professor of medicine at Columbia University Medical Center and the author of the 2010 book “The Emperor of All Maladies: A Biography of Cancer.”

Ang, Vor’s chief executive, said he hopes the company will begin testing its approach in a clinical trial in the first half of next year.

Jonathan Saltzman can be reached at