5AM, JJDC get in on $42M series A round for cell therapy player Vor Biopharma

PureTech’s Vor Biopharma raised $42 million in its series A round, which will support the development of its lead asset, an engineered stem cell therapy for acute myeloid leukemia (AML). The funding comes from 5AM Ventures, RA Capital Management, JJDC, Novartis Institutes for BioMedical Research and Osage University Partners, as well as from PureTech Health.

In addition to pushing Vor’s AML candidate forward, the funding will also build out the company’s pipeline of hematopoietic stem cell, or HSC-based, treatments for blood cancers, according to a statement.

Boston-based Vor came to life in 2016 when it licensed a CAR-T platform from oncologist and Pulitzer Prizewinner Siddhartha Mukherjee, M.D., Ph.D. At the time, Aleks Radovic-Moreno, senior associate of PureTech and co-founder of Vor, told FierceBiotech that he didn’t want Vor to be “following the pack” and become another company to develop a CD-19 CAR-T.

Vor wants to go where CAR-T has seen limited success—though the first two CAR-T therapies scored approval in 2017, there are still been limitations with the technology outside of B-cell leukemias.

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Now, the company is using its engineered HSC platform to tackle challenges in immunotherapy. The HSCs are engineered to generate healthy, functional cells that are protected from specific targeted immunotherapies, the company says.

Vor thinks that combining HSCs with targeted immunotherapies could boost the reach of the latter. It hopes to be able to treat a wide range of patient populations and blood cancers by “enabling new dosing paradigms” that could increase the therapeutic window and improve the safety of such treatments.

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“The need for new therapies for hematologic malignancies is dire. 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,” said Mukherjee, a Vor co-founder and an associate professor of medicine at Columbia University, in the statement.