Vor Biopharma Clinical and Commercial License

RNS Number : 1251U
MaxCyte, Inc.
21 November 2019





Vor Biopharma and MaxCyte Announce Clinical and Commercial License Agreement for Engineered Hematopoietic Stem Cells (eHSCs) to Treat Cancer


-     Clinical and commercial agreement using MaxCyte's recently launched ExPERT™ platform enables up to five of Vor's engineered cell therapies and includes development and approval milestones and sales-based payments

-     Takes MaxCyte's total number of partnered commercial licenses to seven


CAMBRIDGE, Mass. & GAITHERSBURG, Md., (November 21, 2019)-Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, and MaxCyte, Inc., (LSE: MXCT, MXCS), a global cell-based therapies and life sciences company, today announced 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.


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.

About Vor Biopharma

Vor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. By removing biologically redundant proteins from eHSCs, these cells become inherently invulnerable to complementary targeted therapies while tumor cells are left susceptible, thereby unleashing the potential of targeted therapies to benefit cancer patients in need.


Vor's platform could be used to potentially change the treatment paradigm of both hematopoietic stem cell transplants and targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments. A proof-of-concept study for Vor's lead program has been published in Proceedings of the National Academy of Sciences.


Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil. Vor was founded by Dr. Mukherjee and PureTech Health and is supported by leading investors including 5AM Ventures and RA Capital Management, Johnson & Johnson Innovation - JJDC, Inc. (JJDC), Novartis Institutes for BioMedical Research and Osage University Partners.


About MaxCyte

MaxCyte is a clinical-stage global cell-based therapies and life sciences company applying its proprietary cell engineering platform to deliver the advances of cell-based medicine to patients with high unmet medical needs. MaxCyte is developing novel CARMA therapies for its own pipeline, with its first drug candidate in a Phase I clinical trial. CARMA is MaxCyte's mRNA-based proprietary therapeutic platform for autologous cell therapy for the treatment of solid cancers. In addition, through its life sciences business, MaxCyte leverages its Flow Electroporation Technology to enable its biopharmaceutical partners to advance the development of innovative medicines, particularly in cell therapy. MaxCyte has placed its flow electroporation instruments worldwide, including with all of the top ten global biopharmaceutical companies. The Company now has more than 80 partnered programme licenses in cell therapy with more than 45 licensed for clinical use. With its robust delivery technology platform, MaxCyte helps its partners to unlock the full potential of their products. For more information, visit www.maxcyte.com.


This announcement contains inside information for the purposes of Article 7 of Regulation (EU) No 596/2014 (MAR).



Vor Media Contact:

Tom Donovan

(857) 559-3397



MaxCyte Contacts:


MaxCyte Inc.

Doug Doerfler, Chief Executive Officer

Ron Holtz, Chief Financial Officer


+1 301 944 1660

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