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Who leads Vor?
Vor Biopharma was founded in 2016 by PureTech Health and Siddhartha Mukherjee to develop new hematopoietic stem cell (HSC) medicines. The Mukherjee Lab was a leader in engineering HSCs to be protected from targeted immunotherapies. PureTech helped get the group’s IP out of the lab and into a company focused on making new treatments for hematologic malignancies.
What does Vor do?
Vor engineers HSCs to replace the standard-of-care in transplants. The company’s premise is to develop HSCs without CD33, which is a myeloid-derived target in order to make the cells resistant to anti-CD33 medicines. A problem for drugs targeting cells from the myeloid lineage is the lower toxicity threshold versus lymphoid lineage. Vor solves this problem by creating a transplant engineered to be resistant to these drugs. As a result, combinations can potentially dose anti-CD33 drugs much higher. Vor’s HSC platform is meant to be combined with a wide range of immunotherapies from CAR-T cell therapies to ADCs and other biologics. The work that forms the basis of Vor was published in 2019 - https://www.pnas.org/content/116/24/11978
What makes Vor unique?
The key insight of Vor is that there are natural variants in humans where CD33 is knocked out. This implies that using HSCs without CD33 is likely to still be functional in a clinical setting. This allows the company to use new gene editing tools to knockout the gene in HSCs and work to reinvent the bone marrow transplant. I am sure the company is working on extending this theme across different HSC surface targets to increase the dosing of targeted immunotherapies.
With this technology, Vor is first pursuing acute myeloid leukemia (AML), which has a large unmet need and has been resistant to new immunotherapies. For AML patients, Vor is working on replacing their HSCs with their own engineered HSCs (eHSC). Vor is expecting to initiate a phase 1 study of their AML eHSC in 2021, and once they show safety, the company will have the potential to expand into various combination trials. Ultimately, the safety value proposition for AML patients will enable Vor to close several co-development partnerships with companies developing CAR-T cell therapies and biologics in AML.
Why I like what Vor is doing?
AML affects around 60K patients with a little under a third of patients surviving after a bone marrow transplant. More targeted therapies are needed to increase this survival rate; however, myeloid targets often lead to high toxicity levels. For example, Mylotarg, an anti-CD33 ADC for AML patients developed by Pfizer was pulled off the market for some time due to high levels of bone marrow toxicity. With over 80% of AML cells expressing CD33, figuring out a way to kill the target population of cells and sparing normal cells is pretty important for patients. Vor has in vivo data to suggest the eHSCs have the potential to unlock targeted medicines in AML. The next ~2 years will be exciting as Vor enters the clinic.
You can find Vor here.