MaxCyte and National Institute of Allergy and Infectious Diseases to Collaborate on Research for Ultra-Rare Disease Therapy
– Researchers to explore development of new therapies for X-linked chronic granulomatous disease (CGD) using MaxCyte’s gene-correction platform
Maryland, USA – 06 June 2017 – MaxCyte, Inc. today announces it has entered into a Cooperative Research and Development Agreement (“CRADA”) with the National Institutes of Health’s (“NIH”) National Institute of Allergy and Infectious Diseases (“NIAID”) to develop treatments for X-linked chronic granulomatous disease (“CGD”) using next-generation gene correction leveraging CRISPR/Cas9 and MaxCyte’s Flow Electroporation™ Platform.
CGD is an inherited genetic disorder that impairs the function of the immune system and leads to ongoing and severe bacterial infections. The disease affects approximately 1 in 250,000* people worldwide and is currently only treatable through high-risk treatments, such as allogeneic bone marrow transplantation.
NIAID will conduct pre-clinical research evaluating the effectiveness and safety of CRISPR-Cas9 gene editing on models of CGD by “correcting” the faulty gene that causes the disease. MaxCyte will supply mRNA molecules and focus on leveraging its Flow Electroporation™ Platform to develop robust and scalable processes that result in a clinically meaningful correction of mutated gene sequences.
Doug Doerfler, President & CEO of MaxCyte, said: “We are delighted to continue our collaboration with NIAID, one of the world’s leading infectious disease institutes, which is leveraging MaxCyte’s expertise in developing a new generation of genome editing therapy for CGD patients. We believe that this work will validate the use of our platform for developing gene-editing therapies via rapid, cost-effective manufacturing. This agreement, along with recent data announced from a research effort between MaxCyte and NHLBI of NIH in sickle cell disease, further demonstrates our commitment to deliver new therapies to patients where there is an extremely high unmet medical need.”
The MaxCyte/NIAID CRADA marks the latest step in the collaboration between the Company and the NIAID to advance new treatments for CGD, and reflects MaxCyte platform’s ability to be used in multiple fields of indication. MaxCyte received Maryland Stem Cell Research Fund grants in 2015 and 2017 to pursue its collaboration with the NIAID to develop preclinical processes and clinical-scale protocols for CGD and other rare diseases. MaxCyte also presented data generated in the collaboration at the 2015 American Society of Gene and Cell Therapy (ASGCT) annual meeting on the ability of genome editing in hematopoietic stem cells to restore oxidase activity. Earlier this year, MaxCyte shared data from the collaboration at the ASGCT’s annual meeting highlighting the achievement of therapeutic levels of gene correction in hematopoietic stem cells obtained from CGD patients.
MaxCyte is a US-based global company dedicated to driving the acceleration of the discovery, development, manufacturing and commercialization of next-generation, cell-based medicines. The Company provides its patented, high-performance cell engineering platform to biopharmaceutical partners engaged in drug discovery and development, biomanufacturing, and cell therapy, including gene editing and immuno-oncology. With its robust delivery platform, MaxCyte’s team of scientific experts helps its partners to unlock their product potential and solve problems. This platform allows for the engineering of nearly all cell types, including human primary cells, with any molecule, at any scale. It also provides a high degree of consistency and minimal cell disturbance, thereby facilitating rapid, large-scale, clinical and commercial grade cell engineering in a non-viral system and with low-toxicity concerns. The Company’s cell-engineering platform is FDA-accredited, providing MaxCyte’s customers and partners with an established regulatory path to commercialize cell-based medicines. MaxCyte is also developing CARMA, its proprietary, breakthrough platform in immuno-oncology, to rapidly manufacture CAR therapies for a broad range of cancer indications, including solid tumors where existing CAR-T approaches face significant challenges. For more information, visit http://www.maxcyte.com/