10.3.23-Showcase-Osaka-Cellino

WILLIE REAVES: Hello, everyone. I'm Willie Reaves, chief of staff at Cellino, where we are working to make "your cells, your cure," a reality for patients. Cellino is a part of MIT's accelerator that we heard about earlier today called The Engine, and so looking forward to discussing with you the potential for personalized cell therapies.

Cell therapies are leading to cures in their early days, but they're inaccessible to most patients due to the high cost of artisanal manufacturing. So at Cellino, we want to lower the cost of and increase access to cell therapies by dramatically improving the manufacturing process. So Cellino's technology uses induced pluripotent stem cells, or IPSCs, which can be generated from any mature cell type, like a blood or a skin sample. And then those IPSCs can be reprogrammed into whatever cell type is needed to treat a whole variety of diseases.

What we see here are some of the current challenges with manufacturing IPSCs today. So one is that it's manual. A scientist is sitting at the bench, individually evaluating cell colonies to determine which ones are the best.

Two, it's expensive due to these manual processes that really just do not scale and that require a separate clean room for each individualized patient. And third, is there's high variability, because the manual process that we see here is not reproducible from scientist to scientist, and that results in lower cell yields than we could achieve with more automated processes.

Now, this tree shows the incredible potential of IPSCs to be used to treat a whole variety of different diseases, and they span across many different therapeutic areas, many indications, and you can see here with the orange highlights that there are a number of clinical trials going on across the world for a number of these diseases. However, all of these branches and leaves of this tree are all dependent on being able to generate the IPSCs first, because they serve effectively as the base of the tree.

Now, in cell therapy, broadly, there are two primary approaches that are used. One is the autologous, or personalized, approach, where a patient is their own donor, and that's where our tagline of, "your cells, your cure" comes from. And the second is the allogeneic approach, or off the shelf, where one donor cells are used for multiple patients. At Cellino, we lean into the autologous, or the personalized approach, and the reason for that are several.

So one is that it doesn't require immunosuppression. Two is that it's effective for people of all backgrounds, because it doesn't require a donor match, and that can be a challenge for people even within the same family. And three, is because personalized is, ultimately, the safest approach for patients, because there's a very low risk of your immune system rejecting the cells because they are your own cells.

Now, on the left here, you can see one of the automated work cells that we currently have running in our labs at Cellino today. And this automated system is generating patient-specific cells right now. Cellino's platform automates a lot of the manual cell culture techniques that we just saw on the previous slide by using machine-learning algorithms to be able to predict which cell colonies are healthiest to select. And then our laser platform goes in and zaps the cells that are unwanted, leaving the healthy cells there in place.

So this all happens automatically in a single container to reduce contamination risk from manually manipulating cells the way that it's done quite commonly today. And ultimately, we want to move the process from taking up the size of an entire clean room per patient to the size of a cell phone per patient.

Now, on the right here, you see the future system that's currently in prototyping, and each one of these purple boxes is an individual patient sample. So all of these cell processing, the imaging, all of those steps are taking place inside each one of those boxes. And what that would allow us to do is, instead of needing an individual clean room for every individual patient that's in this audience, one of those little purple boxes could do all of the work that's needed to generate a cell therapy that could even potentially be curative for a number of different diseases.

Now, Cellino is thrilled to have an active collaboration with the US. National Eye Institute, which is a division of the National Institutes of Health, and Dr. Kapil Bharti there is leading the US's first clinical trial using autologous IPSCs to develop retinal pigment epithelial cells for the treatment of age-related macular degeneration, a form of vision loss. Dr. Bharti's lab is running comparability studies on IPSCs generated using Cellino's automated platform against IPSCs generated the manual artisanal way that we saw in the videos earlier.

So the goal here is for Dr. Bharti to be able to use Cellino's platform for the later stages of his clinical trial and to be able to do that in a scalable way that's not currently accessible today. Now, we're also in discussions with leading autologous IPSC cell therapy producers, developing cell types for diseases across a whole host of different areas.

Now, thinking about our particular interest at Cellino in Japan and South Korea, the government of Japan has shown significant interest in previous years in advancing regenerative medicine and bio manufacturing, and the PDMA's regulatory processes have really helped to expedite development here. Similarly, South Korea has established a number of biotech zones, including the National Center for Stem Cell and Regenerative Medicine.

And so these government areas of focus are very important when we think about, well, how can we best serve patients? And one of the biggest challenges in Japan and South Korea is the very rapidly aging population, as both countries have the most rapidly aging populations in the world. And many chronic degenerative diseases that could be treated or potentially reversed or cured using IPSC-based cell therapies are particularly debilitating for elderly populations.

So from a partnership perspective, our biomanufacturing system is really going to dramatically accelerate the development and approval of personalized cell therapies. And we're targeting industry and academic collaborations across a number of different cell types and many different disease areas. So we're super interested in enabling scalability for personalized cell therapies for the first time and happy to engage with anyone here. We'll also be outside at the booth.

So again, Willie Reaves at Cellino, where we're working to bring "your cells, your cure" to life. Thank you so much.