MaxCyte and U.S. National Institutes of Health's National Heart, Lung, and Blood Institute Enter Cooperative Research & Development Agreement for Sickle Cell Disease

GAITHERSBURG, Md., June 11, 2018 /PRNewswire/ -- MaxCyte, Inc. today announces it has entered into a Cooperative Research and Development Agreement ("CRADA") with the U.S. National Institutes of Health ("NIH"). Under this new agreement, MaxCyte and the National Heart, Lung, and Blood Institute ("NHLBI"), part of the NIH, will aim to develop treatments for individuals with sickle cell disease ("SCD") using next-generation CRISPR/Cas9-based single-nucleotide correction enabled by MaxCyte's cell engineering platform.

In the search for alternative therapies for SCD, NHLBI will conduct pre-clinical research evaluating the effectiveness and safety of CRISPR-Cas9 gene editing on models of SCD by "correcting" the faulty hemoglobin gene that causes the disease, and addressing DNA mutations in non-corrected cells that contribute to the disease. As part of the agreement, MaxCyte will supply mRNA molecules and focus on leveraging its Flow Electroporation® Technology to develop reliable and effective processes to produce clinically meaningful correction of mutated gene sequences.

This second CRADA with the NIH is further to the announcement on June 6, 2017, where MaxCyte announced a CRADA with NIH's National Institute of Allergy and Infectious Diseases to develop treatments for X-linked chronic granulomatous disease.

Doug Doerfler, President & CEO of MaxCyte, said: "We are delighted to continue our collaboration with NHLBI, one of the world's leading disease institutes, which is leveraging MaxCyte's expertise in developing a new generation of potential treatments for SCD. We believe that this work will further validate our platform for developing gene-editing therapies for a broad range of diseases while enabling rapid, development and commercial manufacturing of new therapies for patients where there is an extremely high unmet medical need."

MaxCyte received a commercialization grant in 2015 and 2017 from the Maryland Stem Cell Research Fund to pursue its collaboration with the NHLBI to develop pre-clinical processes to demonstrate proof-of-biology with MaxCyte's gene correction platform.

Sickle cell disease (SCD) encompasses a group of inherited red blood cell disorders characterized by abnormal hemoglobin (called hemoglobin S or sickle hemoglobin). Approximately 300,000 infants are born with SCD annually worldwide and this number is expected to increase rapidly.ⁱ In addition, the U.S. Centers for Disease Control and Prevention estimate that SCD affects approximately 100,000 Americans, occurring in approximately one in 365 African-American births and one in every 16,300 Hispanic-American births.ⁱⁱ Currently, hematopoietic stem cell transplantation (HSCT) is the only cure for SCD, but many people with SCD are either too old for transplant or do not have a relative with sufficient genetic match to act as a donor.

CRADA # HL-CTCR-18-001

About MaxCyte
MaxCyte is a global cell-based medicines and life sciences company applying its patented cell engineering technology to help patients with high unmet medical needs in a broad range of conditions. MaxCyte is developing novel CARMA therapies for its own pipeline. CARMA is MaxCyte's mRNA-based proprietary platform for autologous cell therapy. In addition, through its core business, the Company leverages its Flow Electroporation Technology to enable its partners across the biopharmaceutical industry to advance the development of innovative medicines, particularly in cell therapy, including gene editing and immuno-oncology. The Company has placed its cutting-edge flow electroporation instruments worldwide, including with nine of the top ten global biopharmaceutical companies, and has more than 50 partnered program licenses in cell therapy including more than 20 licensed for clinical use. With its robust delivery technology, MaxCyte helps its partners to unlock the full potential of their products.

For more information, visit www.maxcyte.com

ⁱ http://www.nejm.org/doi/full/10.1056/NEJMra1510865

ⁱⁱ https://www.cdc.gov/ncbddd/sicklecell/data.html

SOURCE MaxCyte, Inc.

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