Targeted therapies for treating cancer are designed to attack one or more proteins known to be on the surface of cancerous cells—but they don’t discriminate when they come across healthy cells expressing those same proteins.
Vor Biopharma, an oncology company launched by Boston biotech PureTech Health in 2016, has raised $110 million in private financing to test in humans an approach that uses gene editing to facilitate these therapies’ destruction of cancer cells while sparing healthy ones.
The Cambridge, MA-based company says it plans to engineer hematopoietic stem cells so that they don’t have a particular protein that cancer cells are also known to express. Once transplanted, the tweaked cells would create a full range of healthy blood cells. Then the transplant patient would get a therapy, such as a CAR-T or antibody, designed to target the protein the genetic tweaking had removed from the healthy stem cells. If it works, the Vor technology would make the engineered cells “invisible” to the targeted drugs while leaving cancerous cells vulnerable.
“We’re trying to develop all these therapies that only attack cancer and don’t attack the rest of our body, but, of course, we consistently fail to do so,” says president and CEO Robert Ang (pictured), who joined the company in August 2019 from Neon Therapeutics, where he was chief business officer. (Since, Neon has been acquired by BioNTech (NASDAQ: BNTX).)
“There’s been such a large amount of drugs, particularly tackling [acute myeloid leukemia], where these drugs end up attacking your own body, particularly your marrow compartment, much more so than you’d like, and in fact these toxicities can be fatal to the patient or limiting in the utility of these drugs,” Ang says.
To avoid that, Vor’s approach is to preemptively make the bone marrow itself resistant to such treatments. Vor aims to use this approach to first tackle blood cancers, including acute myeloid leukemia (AML). The genetic alternations the company plans to make would get rid of only “biologically redundant” proteins, or those whose functions would continue to be performed even after their removal, the company says. Identifying such proteins is a tricky task, Ang admits.
“We need to make sure that when we cut out expression of these proteins we’re not doing any harm to the cells or to their progeny, and that’s particularly important to hematopoietic stem cells because these are cells that engraft in the patient’s marrow… essentially permanent cells that are there for decades,” he says.
The target the company is moving forward with first—the protein it plans to remove to create its lead candidate, VOR33—is CD33. Ang says the company anticipates moving the investigational therapy into the clinic in the first half of next year.
Vor intends to use the Series B proceeds to pay for costs associated with the VOR33 trials. But Ang says some funds will go toward expanding the biotech’s portfolio as well as finding other proteins that it believes it can safely remove from cells slated for transplant. The company says its approach could improve the safety of cancer treatments including CAR-T cell therapies, antibody drug conjugates, and bispecific antibodies.
PureTech founded the company, which takes its name from a Norse goddess of wisdom, with Columbia University professor Siddhartha Mukherjee, author of “The Emperor of All Maladies: A Biography of Cancer.” The Pulitzer Prize-winning hematologist/oncologist heads the company’s scientific advisory board. In February 2019, Vor raised a $42 million Series A round. Ang says the company, which had six employees when he joined, now has nearly 50.
Joshua Resnick, a managing director at RA Capital Management, in a statement described Vor’s work as an effort to “establish a new standard of care in stem cell transplants.” RA Capital—a crossover investor whose participation in a financing often signals a biotech’s intent to conduct an initial public offering—led Vor’s Series B round. Ang says while the company is always evaluating how to raise capital, it isn’t publicly discussing whether it has plans to pursue an IPO.
Other new backers that participated include Fidelity Management & Research Company, Alexandria Venture Investments, and the Pagliuca family investment office. Steve Pagliuca is co-chairman of Boston-based global private equity firm Bain Capital.
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