Gaithersburg, Maryland – MaxCyte, the global cell-based medicines and life sciences company, today announced that Linghong Li, PhD, Director, Cell Engineering, presented pre-clinical data at the annual meeting of the American Society of Gene and Cell Therapy (ASGCT) in Chicago on Friday, May 18, 2018, in which MaxCyte’s non-viral cell engineering technology was used for CRISPR-mediated gene-correction of a mutation within the hemoglobin gene of cells from a sickle cell disease (SCD) patient.
SCD comprises a group of red blood cell disorders associated with mutations within the gene that encodes hemoglobin. Correction of these mutations via gene editing offers potential for a new curative treatment for individuals living with SCD. Scientists at MaxCyte, the National Institutes of Health’s (NIH) National Heart Lung and Blood Institute (NHLBI) and the NIH’s National Institute of Allergy and Infectious Disease (NIAID) highlighted their most recent in vitro data for the correction of a single-nucleotide SCD mutation in a poster presentation, entitled “GMP-compliant non-viral CRISPR-mediated process correcting the sickle cell disease mutation in SCD patient CD34+ cells achieves 60% wild type adult hemoglobin expression in differentiated erythrocytes.”
Building on their initial in vitro and in vivo successes for gene-correction of HSC from an X-linked CGD patient (DeRavin et al, Sci. Transl. Med. 2017), the researchers took a similar CRISPR-mediated approach for the correction of a mutation in HSC from SCD patients. These latest data demonstrated the extremely high efficiency of MaxCyte’s non-viral cell engineering technology for the safe delivery of the nucleic acids and proteins to SCD patients’ cells necessary for successful CRISPR-mediated gene modification and the subsequent production of therapeutically-relevant levels of adult hemoglobin (HbA) from the corrected cells.
Specifically, their optimized gene correction procedure resulted in 30% to 40% biallelic gene correction of the SCD hemoglobin gene mutation in patients’ HSC, which was maintained following 17 days of in vitro erythroid differentiation. Most significantly, this rate of gene correction led to levels of HbA expression 60% that of wild type cells. In addition, sickle hemoglobin (HbS) production dropped from 100% before correction to 10-20% after correction, potentially conferring additional clinical benefits.
“We’re encouraged that MaxCyte’s technology is at the forefront of enabling non-viral CRISPR-mediated correction of disease mutations at clinically-relevant levels,” said Doug Doerfler, MaxCyte Chief Executive Officer. “We’re excited by these results for their potential to bring therapeutic benefit to individuals living with SCD.”
The abstract can be found at the ASGCT meeting website at annualmeeting.asgct.org/am18/abstracts and the presentation is available on www.maxcyte.com/resource-center/posters-presentations/.
About Sickle Cell Disease
According to the NHLBI, sickle cell disease (SCD) describes a group of inherited red blood cell disorders. Patients with SCD have abnormal hemoglobin, called hemoglobin S or sickle hemoglobin, in their red blood cells. Hemoglobin is a protein in red blood cells that carries oxygen throughout the body. Those who have SCD inherit two abnormal hemoglobin genes, one from each parent. In all forms of SCD, at least one of the two abnormal genes cause a person’s body to make hemoglobin S.
Sickle cell disease is a life-long illness. The severity of the disease varies widely from person to person. In high-income countries like the United States, according to NHLBI, the life expectancy of a person with SCD is now about 40–60 years. Currently, hematopoietic stem cell transplantation (HSCT) is the only cure for SCD. Unfortunately, most people with SCD are either too old for a transplant or don’t have a relative who is a good enough genetic match for them to act as a donor. A well-matched donor is needed to have the best chance for a successful transplant. While there are some treatments that can reduce symptoms and prolong life, more medical options are needed.
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 http://www.maxcyte.com/