Syntax Bio Programs Stem Cells Toward Pancreatic Beta Cells in Four Days
New Cellgorithm™ data presented at ISSCR symposium indicates faster, scalable cell programming and add to Syntax Bio’s
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Syntax Bio, a synthetic biology company programming next generation regenerative medicines, today presented new research showing that its Cellgorithm™ platform differentiated human induced pluripotent stem cells (iPSC) toward pancreatic beta cells in four days. The research indicates that activating the cell’s own genetic instructions in a programmed sequence could enable faster, more efficient and scalable production of cell therapies, including a potentially transformative treatment for people with type 1 diabetes.
The findings were shared in an oral presentation and poster at the International Society for Stem Cell Research (ISSCR) iPSC 20th Anniversary Symposium, “20 Years of iPSC Discovery: A Celebration and Vision for the Future,” held Oct. 20-22 at the Kyoto International Conference Center in Kyoto, Japan.
Cellgorithm uses CRISPR activation (CRISPRa) to switch on the cell’s own genes without cutting or editing the genome. It turns genes on in a specific order and at carefully controlled times, mimicking how specialized cells form during human development. A single delivery of transient DNA instructions guides the cells through the programmed sequence.
Researchers tested over 170 Cellgorithm variants across three screens, drawn from 29 pancreatic lineage genes, with each variant activating up to 10 genes in five or fewer timed steps. Single-cell RNA sequencing and protein expression analyses showed that specific cells were guided toward endocrine precursor and pancreatic beta-like cell states within four days. The resulting cell states depended on which genes were activated and when. Other efforts created “gridlocks,” stalling cells at intermediate stages even when instructions for later stages were included.
As the technology is applied across more cell states, each program generates new data that maps the human cell lineage tree and provides insight into how cells respond to specific genetic instructions. These compounding data feed back into the platform, improving its ability to predict efficient paths to desired cell states and to generate each new cell state faster than the last.
“These findings show that Cellgorithm can mimic how stem cells develop and reveal which genes move that process forward or hold it back,” said Ryan Clarke, PhD, co-founder and chief scientific officer of Syntax Bio. “In this study, we directed stem cells toward pancreatic beta cell states in just four days. Learning how to precisely control that process could help overcome the longstanding bottlenecks that have made producing regenerative medicines slow and labor-intensive, enabling faster, more reliable and scalable production. What we learn applies to every cell type we program next.”
Syntax Bio also completed Aim 1 of its pancreatic beta cell program, supported by Breakthrough T1D through its Industry Discovery and Development Partnership (IDDP). Aim 1’s milestones included screening and selecting Cellgorithm candidates.
The company is now advancing Aim 2, which is focused on optimizing the selected Cellgorithms and developing the processes needed to scale manufacturing, alongside functional and preclinical evaluation in collaboration with Mayo Clinic.
“Completing these screening milestones advances our lead beta cell program into optimization and process development,” said John Craighead, PhD, CEO of Syntax Bio. “The findings presented in Kyoto reinforce Cellgorithm’s broader potential and bring us closer to our vision of programming every human cell state at scale. With support from Breakthrough T1D and in collaboration with Mayo Clinic, we are building toward a transformative cell therapy for people with type 1 diabetes.”
Syntax Bio is collaborating with Mayo Clinic to evaluate pancreatic beta cell therapies. The collaboration combines the Cellgorithm platform with Mayo Clinic’s expertise in pancreatic biology, preclinical diabetes models and clinical translation.
The ISSCR iPSC 20th Anniversary Symposium convenes the global stem cell community in Kyoto, the birthplace of iPSCs, to mark 20 years since Nobel laureate Shinya Yamanaka’s discovery and explore the future of iPSC research and clinical applications.
Syntax co-founder and chief scientific officer Ryan Clarke delivered the oral presentation, “Cellgorithms: Programming Human iPSC Differentiation With Modular, Time-Resolved CRISPRa Cascades,” on Wednesday, Oct. 21, 2026. Clarke also presented the poster with the same title (Poster 288).
Mayo Clinic has a financial interest in the technology referenced in this press release. Mayo Clinic will use any revenue it receives to support its not-for-profit mission in patient care, education and research.
About Syntax Bio
Syntax Bio is a synthetic biology company programming next generation regenerative medicines. Its proprietary Cellgorithm™ platform uses a CRISPR-based system to program and accelerate stem cell differentiation, transforming how cell therapies are discovered, developed and manufactured. By replacing slow, labor-intensive differentiation processes with a rapid, programmable approach that mimics human development, Syntax Bio can generate specialized cells in days or weeks rather than months, more reliably and at scale, unlocking new possibilities across the biotechnology industry. Learn more at syntax-bio.com.
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