Mission Bio's New Translocation Analysis Offers Insights Into Critical Safety Assessment for Gene-Edited Cell Products

Mission Bio recently announced the launch of a new single-cell capability that enhances our understanding of translocations (chromosomal rearrangements) in gene-edited therapies. Measuring predicted translocations along with on- and off-target editing at single-cell resolution can enable developers to design more efficient and safer therapeutics. Data being presented at the Annual American Society of Gene & Cell Therapy (ASGCT) Meeting further validates Tapestri’s translocation analysis for comprehensive characterization of genome-edited cells, which is crucial for the development of cell-based therapies like CAR-T cells and stem cell-based therapies.

“Conventional bulk methods for detecting chromosomal rearrangements are limited in scale and accuracy,” said Vanee Pho, PhD, Senior Director of Product Management, Cell and Gene Therapy at Mission Bio. “Through our single-cell Tapestri Platform, our latest translocation analysis provides our customers with a comprehensive picture of gene edits and unwanted chromosomal aberrations at single-cell resolution — enabling critical safety assessment of these innovative therapies. Our hope is to provide biomedical researchers with a definite picture of these drug products and to streamline the clinical development process.”

Gene editing technologies like CRISPR hold the potential to treat previously incurable diseases by correcting disease-causing mutations. However, CRISPR can also lead to off-target edits due to the inadvertent cutting of the genome beyond the intended target sites, resulting in incorrect insertions and deletions (indels) or structural variants such as translocations, which have been linked to the development of cancer. Confident detection of off-target effects and structural variants presents a significant safety challenge and a bottleneck for the development of CRISPR-based therapeutics.

“CRISPR genome editing offers a way to finally eradicate genetic disorders, providing hope for patients with limited effective treatment options,” said Dr. Ayal Hendel, a pioneer in CRISPR-based therapeutics and Principal Investigator at the Bar-Ilan University in Israel. “With Mission Bio’s translocation approach, we can profile off-target activity and genome-editing efficiency more robustly and with unparalleled precision. This advancement paves an accelerated path to deliver life-saving medicines, utilizing CRISPR to restore the immune system in immunodeficiencies and blood disorders.”

In an oral presentation ASGCT, Dr. Hendel will highlight data validating Tapestri’s translocation analysis in T-cell CRISPR editing. More specifically, Dr. Hendel will describe the usage of the Tapestri single-cell DNA sequencing (scDNA-seq) platform to fully profile CRISPR-editing outcomes in human primary T-cells, including translocation identification.

The addition of translocation analysis is one of several updates to the Tapestri Genome Editing Solution and includes the recently released antibody hashing capabilities that enable sample multiplexing, allowing drug developers to increase sample throughput while reducing labor and reagent costs associated with single-cell sequencing. The Tapestri platform also measures known DNA translocations for hematological-oncology applications via Tapestri custom panels.

Mission Bio is a leading life science company, specializing in the advancement of single-cell DNA and multiomic analysis. The company’s Tapestri Platform is unique in its capabilities, offering an unparalleled level of granularity and precision that is critical for complex research areas such as cancer studies, pharmaceutical development, and advanced cell and gene therapies. Unlike traditional methods such as bulk sequencing, Tapestri provides a level of precision that opens the door for more tailored and effective treatment strategies. Researchers globally depend on Tapestri to identify rare cell populations, understand mechanisms of therapeutic resistance and response, and establish key quality metrics for next-generation medical treatments. Founded in 2014, Mission Bio has secured investment from firms including Mayfield Fund, Novo Growth, Cota Capital, and Agilent Technologies. With the Tapestri Platform, Mission Bio is setting the standard in the field, contributing significantly to the progress of personalized medicine and targeted therapies. For more information, visit missionbio.com.