MaxCyte, Inc. and National Institute of Allergy and Infectious Diseases Announce Pre-Clinical Results from Collaborative Study
January 12, 2017 at 12:30 pm IST
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MaxCyte, Inc. announced pre-clinical results from a collaborative study with investigators at the National Institutes of Health's (NIH) National Institute of Allergy and Infectious Diseases (NIAID). Results from the study demonstrated clinically relevant levels of correction in the CYBB gene mutation in long-term engrafted hematopoietic stem cells (HSC) and differentiated neutrophils in cells obtained from individuals with X-linked chronic granulomatous disease (X-CGD). Using MaxCyte's patented Flow Electroporation Technology, researchers demonstrated that transfecting three molecules, a single-strand oligonucleotide correction template, along with messenger RNA encoding for CRISPR-Cas9 gene editing complex and selected guide RNA into HSC obtained from individuals with X-CGD, resulted in correction of mutation in the CYBB gene. This correction occurred at clinically relevant levels following long-term engraftment in preclinical models. 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 one in 250,000 people worldwide, according to MedScape, and is currently only treatable through high-risk treatments, such as allogeneic bone marrow transplantation. It is caused by a mutation in the CYBB gene, that enables immune cells to defend against microbes. The MaxCyte and NIAID researchers were able to restore function of the immune cells by repairing this mutation in CYBB.
MaxCyte, Inc. is a commercial cell engineering company. The Company is focused on providing enabling platform technologies to advance the discovery, development and commercialization of cell therapeutics, including cell and gene therapies, and to support cell-based research and development. It has developed and commercialized its proprietary Flow Electroporation technology, which is used by biopharmaceutical companies in the complex engineering of a variety of cells. Its ExPERT platform is based on its Flow Electroporation technology, which has been designed to address this rapidly expanding cell therapy market and can be utilized across the continuum of the cell therapy sector, from discovery and development through commercialization of cell-based medicines. The ExPERT family of products includes four instruments, which are known as the ATx, STx, GTx and VLx, as well as a portfolio of proprietary related disposables and consumables.