Anjali Pradhan, MS, on Using Single Cell Sequencing for Gene Editing Quality Control

“What we've seen is in the industry today there's a lot of excitement with new editing technologies... But what you are always concerned about is what is the safety of those edits? How can you be sure that your edit is as it was intended to be and if it's going in the right place? That's a huge concern to doctors, especially with various editing efficiencies, etc. How do you make sure you're doing the right thing?”

Gene editing approaches to treating both rare and common diseases are rapidly gaining interest among biotech companies, academic institutions, and the medical field in general. A wide range of different methods for gene editing, such as CRISPR-based editing and prime editing, are being researched and a number of these methods are currently being evaluated in clinical trials or preclinical research. On the whole, gene editing methods have the potential to provide a transformative or even curative impact on patient’s diseases. That said, gene editing as a modality also carries potential safety risks and barriers to efficacy. As such, quality control (QC) is of critical importance as these novel therapeutics move into the clinic to ensure that the intended edits to the genome are made in a precise manner and that unintended changes to the genome are avoided.

Mission Bio is seeking to address this need with the use of its Tapestri Genome Editing Solution, which uses DNA sequencing to detect gene edits at a single cell level. Anjali Pradhan, MS, the chief product officer at Mission Bio, recently presented a poster on the company’s platform at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD. CGTLive® interviewed Pradhan at the conference to learn more. Pradhan explained the need for QC when applying gene editing approaches and spoke about how the company’s technology is intended to address this need.

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