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MaxCyte and U.S. National Institutes of Health’s National Heart, Lung, and Blood Institute enter cooperative research & development agreement for Sickle Cell Disease
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.
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.