Brian Kim, MBA, on the Importance of Single Cell Analysis to Cell and Gene Therapy

“I think what physicians and caretakers will ultimately care about is whether or not the gene editing drugs that go into patients will be both safe and efficacious... Our platform allows us to be able to characterize those important measures around safety and potency for our drugs.”

Sequencing of DNA in collections of cells can provide many different types of useful information for a variety of applications. For example, it can provide information about the presence of certain mutations related to cancer in a sample of cells. In the context of cell and gene therapy specifically, DNA sequencing is an important tool for characterizing the efficacy and safety of a therapeutic product. For example, it can reveal whether the desired genetic change has been made and whether unintended genetic changes, which could potentially constitute safety risks, have occurred.

Traditionally, DNA sequencing has been carried out with bulk measure sequencing. This approach is limited, however, because it can’t provide precise information about the cooccurrence of changes or mutations within specific, single cells within the larger bulk product. Single cell sequencing, a more advanced approach to DNA sequencing, overcomes this limitation, providing the ability to observe changes at a single cell level. Standard approaches to single cell sequencing are still held back by limitations on their time efficiency and cost efficiency, with assays for different types of changes, such as translocations or off and on target edits, having to be run separately.

Mission Bio is seeking to overcome this limitation through the use of its Tapestri platform for single cell sequencing. The company is developing a suite of assays for different points of interest relevant to cell and gene therapy products that can all be run together through the Tapestri platform.

CGTLive® spoke with Brian Kim, MBA, the chief executive officer of Mission Bio, at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD, to learn more. Kim explained the advantages of single cell sequencing, and of the Tapestri platform more specifically, and also discussed the company’s future plans to launch additional assays and potentially further cut down on the cost-per-sample for the platform.

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