Mission Bio launches genome editing solution for Advanced Therapies

Designed to meet the pressing need for high-resolution analysis of genome editing, the company said this first-of-its-kind solution addresses a long-standing gap in advanced therapy development, disease modeling, and functional genomics.

The technology was initially previewed at The American Society of Gene and Cell Therapy annual conference.

The Tapestri Genome Editing Solution has numerous applications for genome engineers, advanced therapy developers, and disease modelers.

According to Mission Bio, the solution allows for the rapid characterization of gene-edited drug products, identifying on and off-target editing, the zygosity of edits, and the co-editing of multiple targets in single cells.

The solution also supports multi-omic analysis, which can paint a clearer picture of cell engineering, as one can determine which cell types are edited, or perhaps validate a knockout through the loss of a protein.

Gene editing technologies, such as CRISPR, have offered immense opportunities for addressing incurable and untreatable diseases like sickle cell disease, where the first CRISPR-edited cell therapy may be approved by the U.S. Food and Drug Administration later this year.

Gene editing has also proven to be a powerful strategy in developing disease models and determining genomic drivers of disease progression. However, they also bring intricate challenges such as evaluating the fidelity of both intended and unintended edits.

Conventionally, researchers have to perform single-cell cloning in order to fully understand what types of edits were occurring in individual cells, a labor intensive process that involves cell culture for several weeks. 

Mission Bio’s Tapestri was developed to provide a high-throughput workflow that combines genotypic and immunophenotypic assessments in single cells for samples of thousands of cells, capturing details often overlooked by bulk analysis methods.

The platform also features automated data reporting, eliminating the need for devoted bioinformatics resources and saving days of computational work.

Brittany Enzmann, product manager for Mission Bio’s Tapesti, said: “The Tapestri Genome Editing Solution equips researchers with the capability to scrutinize gene therapies at the single-cell level, a crucial step for ensuring patient safety. By providing this unparalleled resolution, we’re not just offering a new tool; we’re contributing to the evolution of safer and more effective gene therapies. The result is a faster transition from the lab to the patient.”

Saar Gill, associate professor of medicine at the University of Pennsylvania, added: “This multi-omic genome-editing solution will enable critical insights for gene-edited cell therapies beyond what conventional bulk analysis can offer. The automated report gives an immediate and intuitive first look at our data, something that would usually require hours of bioinformatic labor. All in all, we are better equipped to understand our editing results with this technology.”

The technology will be showcased at the upcoming Cell Therapy Analytical Development Summit in Amsterdam from September 19-21.