Vanee Pho, PhD, on Detecting Translocations in Gene Edited Cells

“[S]ingle cell [analysis] takes us an extra step forward in understanding down to the actual number of cells that have these translocations, which is something bulk techniques cannot show.”

Gene editing approaches to genomic medicine, such as CRISPR/Cas9-based gene editing therapies, hold tremendous potential to treat a wide variety of rare and common diseases by allowing for precise editing of genes of interest through targeted double-strand breaks.¹ Although, gene editing approaches are not perfect, and unintended changes to the genome, such as double-stranded breaks in the wrong location and translocations, can occur. Such changes can carry a number of risks, and translocation in particular, also known as chromosomal rearrangement, has been linked to cancer and other harmful effects. As such, companies developing gene editing therapies need to invest substantial time and resources into detecting such changes when evaluating their product candidates in order to ensure optimal safety. Although DNA sequencing carried out with bulk measure sequencing can detect translocations, it has limitations in terms of scale and accuracy. Mission Bio is seeking to address these limitations through its Tapestri Genome Editing Solution, which the company recently updated with a new translocation analysis feature. The company recently shared information about this update at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD.

At the conference, CGTLive® spoke with Vanee Pho, PhD, the senior director of product management, cell and gene therapy, at Mission Bio, to learn more. Pho discussed the importance of detecting translocations caused by gene editing products and how Mission Bio’s technology can help address this need. She also spoke about a presentation given at the conference by Ayal Hendel, PhD, the principal investigator at the Bar-Ilan University in Israel, with whom Mission Bio is working on research related to characterizing off-target genetic changes resulting from CRISPR-based gene editing.²

Click here to view more coverage of the 2024 ASGCT Annual Meeting.

REFERENCES
1. Mission Bio’s new translocation analysis offers insights into critical safety assessment for gene-edited cell products. Mission Bio. April 25, 2020. Accessed June 6, 2024. https://missionbio.com/press/translocation-analysis/
2. Hendel A. Precise measurement of CRISPR genome-editing outcomes through single-cell DNA sequencing. Presented at: ASGCT Annual Meeting 2024, May 7-10; Baltimore, Maryland. Abstract #254