On November 21, 2019 Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, and MaxCyte, Inc., a global cell-based therapies and life sciences company, reported a clinical and commercial license agreement under which Vor will use MaxCyte’s Flow Electroporation technology to produce eHSCs and initiate Investigational New Drug (IND)-enabling studies to accelerate its progress towards the clinic (Press release, Vor BioPharma, NOV 21, 2019, View Source [SID1234551588]).
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Under the terms of the agreement, Vor obtains non-exclusive clinical and commercial use rights to MaxCyte’s Flow Electroporation technology and ExPERT platform to develop up to five engineered cell therapies, including VOR33, Vor’s lead eHSC candidate, which is in development for acute myeloid leukemia (AML). In return, MaxCyte will receive undisclosed development and approval milestones and sales-based payments in addition to other licensing fees.
Vor will use MaxCyte’s cell engineering platform to deliver its gene editing machinery into hematopoietic stem cells to remove biologically redundant cell surface proteins that are also expressed on blood cancer cells. Once the eHSCs are transplanted into a cancer patient, these cells are effectively hidden from complementary targeted therapies that target the relevant protein, while diseased cells are left vulnerable to attack. Vor’s approach thereby could unleash the potential of targeted therapies by broadening the therapeutic window and improving the utility of complementary targeted therapies.
"MaxCyte is a leader in GMP electroporation technology, and we are thrilled that this agreement provides us with long-term access to a platform technology applicable to a pipeline of eHSC programs used to treat AML and other blood cancers," said Sadik Kassim, Ph.D., Chief Technology Officer of Vor. "As we build on promising in vivo data from our lead candidate VOR33, we can now expand our manufacturing capabilities to support later-stage studies, regulatory filings and commercialization of VOR33."
MaxCyte’s ExPERT instrument family represents the next generation of leading, clinically validated, electroporation technology for complex and scalable cellular engineering. By delivering high transfection efficiency with enhanced functionality, the ExPERT platform delivers the high-end performance essential to enable the next wave of biological and cellular therapeutics.
"We look forward to expanding our relationship with Vor Biopharma as the company pioneers a potential future standard of care in hematopoietic stem cell transplants for cancer patients in need," said Doug Doerfler, President & CEO of MaxCyte. "This agreement represents another key business milestone for MaxCyte, emphasizing the value of our technology platform applied to next-generation engineered cell therapies that may make a true difference in patient outcomes."
About VOR33
Vor’s lead product candidate, VOR33, consists of engineered hematopoietic stem cells (eHSCs) that lack the protein CD33. Once these cells are transplanted into a cancer patient, CD33 becomes a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. In so doing, Vor aims to improve the therapeutic window and effectiveness of CD33-targeted therapies, thereby potentially broadening the clinical benefit to patients suffering from AML.