Cell therapies are rapidly becoming a potent weapon in our medical armory, with existing treatments offering hope for patients with certain cancers and rare diseases. However, as more prevalent conditions enter the cell therapy research frame, concerns grow over the prohibitive costs of these treatments, often running in the multiple millions per dose. Ryan Clarke, co-founder and CEO of Syntax Bio, acknowledges these cost concerns as unsustainable for our healthcare system.
Several initiatives have emerged with a shared goal of improving cell therapies manufacturing techniques. Ranging from fully automated process with robotics and software to the application of gene-editing technologies to donor cells, diverse solutions exist.
Syntax Bio harnesses induced pluripotent stem cells (iPSCs)—capable of transforming into various types of cells—for their research. They apply the CRISPR gene editing tool, guiding iPSCs through multi-step processes that convert them into a specific cell type.
Since its launch in 2021, Syntax Bio has proven its technology can convert iPSCs into four diverse cell types and has even clinched the winner position in the biopharma track at the 2023 MedCity News INVEST conference. The firm continues to make strides in technological progress and is currently looking to secure $20 million in Series A financing.
The platform technology roots at the University of Illinois Chicago, where Clarke’s PhD research centered on the transcription factors of genes controlling stem cell differentiation. Later joining Sana Biotechnology, a firm developing new cell and gene therapies, Clarke was drawn to the prospects of commercializing the technology he had constructed during his graduate school days.
Current methodologies to create cell therapies from stem cells include directed differentiation and synthetic biology approach. Directed differentiation, though capable of creating any cell type, is slow, expensive, and difficult to upscale. The synthetic biology approach, while faster, more scalable and less costly, has limitations due to its applicability on certain cell types and skips critical intermediate steps.
Syntax Bio has engineered its cell therapy technology to overcome the limitations observed in both directed differentiation and synthetic biology methods. The technology, named ‘Cellgorithms’ by Syntax, uses episomal plasmids that leverage CRISPR to regulate genes in a specific sequence, directing the iPSC to the final cell state in a process taking approximately a week.
A study detailing the Cellgorithm system was published in the Molecular Cell journal in 2021. This early-stage research secured $9 million in seed financing, co-led by DCVC. Syntax Bio’s CEO states that they are now at the technology’s third generation, which has seen improvements in efficiency and modularity.
Syntax targets partnerships with larger companies potentially leading to human testing of new cellular therapies. Simultaneously, it aims to collaborate with stakeholders across the cell therapy ecosystem, including drug developers and contract development manufacturing organizations. The firm can adapt its technology with that of partner companies to support large-scale cell manufacture.
Syntax’s potential extends to generating pancreatic beta cells, offering a possible solution for type 1 diabetes cell therapy. Despite companies like CellTrans having received FDA approval for a type 1 diabetes cell therapy and giants like Vertex Pharmaceuticals progressing with their programs, Syntax’s vice president of corporate strategy & ops, Leo Kelly, prefers to view these entities as potential clients rather than competitors.
As Syntax continues leveraging CRISPR to revolutionize cell therapy manufacturing, it exemplifies a promising avenue in healthcare technology. Its scalable and cost-effective techniques could spur further breakthroughs, transforming the prospects for patients around the world.