MaxCyte and NIH Office Partner to Develop Cell Therapy for Sickle Cell Disease

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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MaxCyte, a company specialized in cell-based medicines, announced it has entered in a research agreement with the National Heart, Lung and Blood Institute (NHLBI) to develop potential therapies for sickle cell disease (SCD).

Researchers will use MaxCyte’s gene-correction platform, Flow Electroporation Technology, in their work. The platform is a next-generation CRIPSR/Cas9-based corrector of single-nucleotides, the building blocks of nucleic acids (DNA or RNA).

MaxCyte will provide messenger RNA (mRNA) molecules and focus on leveraging technology to develop ways to correct mutated gene sequences. mRNA conveys genetic information from DNA to the parts of a cell involved in the process of protein production.

The U.S. institute, part of the National Institutes of Health, will be responsible for conducting preclinical research evaluating the safety and efficacy of CRISPR-Cas9 gene editing on SCD models. This approach aims to correct the faulty hemoglobin gene that causes sickle cell disease.

“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 … where there is an extremely high unmet medical need,” Doug Doerfler, president and chief executive officer of MaxCyte, said in a press release.

The partnership was announced in advance of June 19, World Sickle Cell Disease Day.

This is the second grant given MaxCyte by the Maryland Stem Cell Research Fund to collaborate with the NHLBI on ways to demonstrate proof-of-biology with MaxCyte’s gene correction platform. The first grant was awarded in 2015.

MaxCyte announced in May that its technology platform had corrected a SCD-associated mutation in a patient’s hematopoietic stem cells (HSCs), a type that give rise to blood cells.

The work resulted in 30 to 40 percent gene correction of the hemoglobin gene mutation. The correction was maintained for 17 days, the time required for HSCs to transform into red blood cells, a process called differentiation. In these differentiated cells, the gene correction also increased the levels of hemoglobin expression, up to 60 percent of the levels seen in healthy red blood cells, the company reported.

Study data were presented in a poster at the 2018 American Society of Gene and Cell Therapy meeting in Chicago.