MaxCyte’s in Demonstrates CRISPR-Cas9 X-linked CGD gene repair study

MaxCyte has been engaged in a study with National Institutes of Health’s (NIH) allergy and infection section to show certain CYBB gene behaviour in patients with X-linked chronic granulomatous disease (X-CGD).

The tech supplier for pharmaceutical companies on Thursday said that the results that were published in Sciente Translational Medicine were encouraging.

In the publication entitled: “CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease,” it was showen how MaxCyte’s proprietary was effective, how high-performance technology can correct genetic mutations, especially in cases where traditional viral vector-based gene insertion methods have not been effective.

Using MaxCyte’s 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.

Madhusudan V. Peshwa, Ph.D., MaxCyte’s Chief Scientific Officer (CSO), said that the demonstrates the commitment of MaxCyte to fostering development of novel cell therapies and supporting its partners’ use of MaxCyte’s delivery platform. “MaxCyte invests early in translational development of non-virally engineered and ex vivo gene-edited cell therapy products,” said MaxCyte’s CSO.

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.