Driving Innovation in Cell Therapy Manufacturing with Ryan Clarke Syntax Bio

 Ryan Clarke, Co-Founder and CTO of Syntax Bio, is focused on solving manufacturing challenges in producing stem cell-derived therapies, specifically the process of stem cell differentiation. The Cellgorithm platform was designed to make differentiation an engineering problem by using a modified form of CRISPR to turn genes on and off in a specific, controlled sequence, reducing processing time from months to days and resulting in cost savings. One goal is to use AI and machine learning to build models capable of predicting optimal gene regulation sequences accelerating the discovery of new differentiation protocols and treatments for degenerative diseases.

Ryan explains, "The key problem that we are focused on applies to stem cell-derived cell therapies. And so, just for a little bit of context setting, we use induced pluripotent stem cells or IPS cells. And about 20 years ago, when these were first derived, everybody was very excited because this is the platform where you could then have an infinite supply of stem cells to then make any tissue-specific cell type at will, theoretically. Fast forward 20 years, and there are finally some cell therapies in phase three clinical trials, but none are approved yet. And that just tells you that the development cycle for this modality is actually slower than the other modalities, like biologics or small molecules. And the problem is manufacturing in particular, or how do you convert the stem cell into the target tissue cell type for the process of stem cell differentiation? And so we are solely focused on making stem cell differentiation an engineering problem rather than a kind of dark art."  

"We have a program for type one diabetes that is making pancreatic beta cells from IPS cells. And we have also done some work in the musculoskeletal system. So these other cell types we focus on are more demonstrations of the platform technology, but we are interested in possibly co-developing those with other pharmaceutical partners, and that's the musculoskeletal cells of the hematopoietic system. And we've done some work on retinal cells as well, but we endeavor to make many other cell types. And our goal is to partner with the therapeutic experts in the area to develop these."

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