Multiomic Single-Cell Sequencing and the Ebbs and Flows of the Gene Therapy Field

Brian Kim, MBA

Mission Bio, a company seeking to overcome limitations of bulk sequencing and standard single cell sequencing methods through the use of a unique platform, gave several presentation related to its technology at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2024, in New Orleans, LA. CGTLive® sat down with Brian Kim, MBA, the chief executive officer of Mission Bio, at the meeting to get his insight on the key takeaways of what the company brought to the conference this year.

Kim highlighted several of Mission Bio's presentations and discussed what the company is working on more generally, noting several collaborations. He also touched on the state of the field in general and expressed optimism for its trajectory in the future.

CGTLive: Can you give some background about Mission Bio?

Brian Kim, MBA: Mission Bio is a single cell multiomic platform that is used for mostly clinical applications. It differentiates itself from other single cell companies in that it works with DNA and looks at proteins. Our platform is really used for customers who aren't necessarily trying to discover new things, which is a lot of what current single cell platforms are being used for, but [instead for] once you've discovered it and you know what you're looking for, to really validate it for either therapeutic purposes or for diagnostic purposes.

Can you give an overview of what the company is presenting at ASGCT this year?

We have a couple presentations here. One is with the work that we have done with Genentech, and another is a really important study that Ayal Hendel, PhD, has worked on with us. They're going to be, I think, really very exciting developments for our customers. One of the other big developments that we will have announced here at ASGCT is a partnership with Integrated DNA Technologies (IDT). This actually was a partnership that actually originated at ASGCT in 2024 in Baltimore. Essentially what we are doing is we are incorporating IDT's world-class rhAmpSeq technology into our assay. What this will effectively do is it will allow us to create a very sensitive assay that will look for on-target efficacy and off-target detection to really help characterize the safety of CRISPR-based medicines for our customers.

How would you summarize the big picture implications you would want the broader healthcare community to take away from Mission Bio's presentations this year?

I think this is an interesting time we're living in right now and cell and gene therapy is going through a transition period right now, but I think it's really important to note that it's a transition period. I don't think people should really lose too much sleep that this is going to go away. A lot of drug modalities and technologies have these ebbs and flows, but we just saw this morning there was an announcement by the New York Times that a little baby was saved with a CRISPR-based therapeutic for a very rare genetic disorder that this child had. When you see things like that happening with patients with the therapies that we have right now, you know that it's going to go someplace in the future.

So as it relates to ASGCT and with Mission Bio we are absolutely committed to this space. We are continuing to develop our assays and our products really make an impact to our customers. To kind of give you a sense of it, our company is or has been involved in over 17 clinical trials for biopharma customers and our products right now are being adopted by 3 commercial and academic labs to be used as a laboratory-developed test. And so for us, we're committed to this space and we are in here for the long run because we know this momentary issue that we're facing will eventually turn the other way.

Are there any future plans for Mission Bio that you can discuss a bit?

I've been in post as the CEO of this company for about almost 18 months, and since I've been in place I've really focused on 4 key things: Revenue growth, continuing to innovate great products, clinical utility for our products, and really just continuing to optimize and really run a very optimized business performance and operations. I'm happy to note that revenue-wise, we are going to have a really strong year this year because of a lot of the work that our teams have been taking on. I'd say, from an innovation perspective, we are really being focused on the things that really matter to our customers. One of the big innovations that we will be announcing very soon is the incorporation of targeted gene expression into our assay. Right now, we work with DNA to get genotyping, we can detect proteins for immunophenotyping, and we'll now be able to incorporate into our assay RNA for targeted gene expression. We think that ability to analyze all these analytes for our customers is going to be very important. That's what our customers have been asking for from us for some time so we're really thrilled about being able to deliver this into the marketplace.

As it relates to clinical utility, we're also really thinking and taking a hard look at where within diagnostics our platform will have the most relevance. We announced at the beginning of late 2024 a significant collaboration that we're doing with the University of Miami.

University of Miami and C. Ola Landgren, MD, PhD, are world renowned for their work in treating multiple myeloma. The work we're doing with them is focused on how we can use our assay to actually help guide therapeutic selection after a patient relapses and have exhausted their frontline treatments. This is something that we're working with them on right now to actually collect the data, and we'll be able to say something more about this towards the end of the year. But if we can collect the data that actually shows that with our platform we can guide the therapy that our patients who have exhausted frontline therapies should go on, we think that this is going to be really meaningful to our customers. So that's another example of collaboration and working with the external world to really prove the clinical utility of our platform.

This transcript has been edited for clarity.

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