Mesenchymal stem/stromal cells (MSCs) are emerging as a promising tool in cell therapy due to their strong immunomodulatory and anti-inflammatory properties, their capacity for tissue regeneration, and a favorable safety profile. Unlike other cell-based therapies, such as CAR T cells that may trigger severe immune responses like cytokine storms, wild-type MSCs have shown no such adverse reactions when administered to humans. However, the widespread clinical application of MSCs has been limited by challenges in producing therapeutically effective cells consistently and at scale. To date, only one MSC-based therapy has been approved by the U.S. Food and Drug Administration (FDA).
A key obstacle in developing MSC therapies is the ongoing requirement for new tissue donations from various donors. This dependence leads to high variability and restricted batch sizes due to the limited expansion capacity of each donor-derived sample.
To address these limitations, the University of Wisconsin–Madison and Cynata Therapeutics developed the Cymerus™ platform. This technology leverages induced pluripotent stem cells (iPSCs) to enable scalable and reproducible manufacturing of MSC-based therapies. Dr. Mathias Kroll, Chief Business Officer at Cynata, provided insights into the Cymerus platform and the broader potential of MSC therapies, especially for under-treated conditions like osteoarthritis.
MSCs naturally exist in the body and are known for their anti-inflammatory, immunomodulatory, and tissue-repairing functions. These cells can differentiate into other cell types, aiding in the replacement of damaged or dysfunctional tissue. As a result, MSCs have been extensively researched in regenerative medicine.
While several companies have developed MSC-based therapies using donor-derived methods, Cynata’s Cymerus platform offers distinct advantages by applying a scientifically rigorous approach that supports large-scale production.
Cymerus is a proprietary process that creates MSCs from iPSCs, offering a stable and reproducible solution that solves the scalability issue that has hampered the field. iPSCs can be expanded almost indefinitely, which allows for the production of large, consistent batches of MSCs.
Traditional methods of MSC production involve harvesting cells from bone marrow or other tissues—a process that is invasive and typically yields a limited number of cells. To achieve pharmaceutically relevant doses—often hundreds of millions of cells per patient—requires extensive culture expansion. However, excessive expansion can lead to cell senescence and reduced therapeutic function. This makes donor-derived MSCs more suited for niche, low-volume applications.
In contrast, iPSC-derived MSCs from the Cymerus platform are produced in a way that maintains cell potency and avoids exhaustion. This enables large-scale application of MSC therapies, potentially serving widespread diseases like osteoarthritis more effectively.
The recent FDA approval of a donor-derived MSC therapy for treating steroid-refractory acute graft-versus-host disease (GVHD) in pediatric patients underscores the growing acceptance of MSCs as viable therapies. This regulatory success has strengthened Cynata’s confidence in their approach.
Cynata is currently conducting a Phase 3 clinical trial for osteoarthritis, in collaboration with Professor David Hunter at the University of Sydney. The trial builds upon earlier promising studies using tissue-derived MSCs. The goal is to evaluate the extent to which Cymerus-derived MSCs can reduce inflammation, regenerate tissue, and potentially slow or halt the progression of osteoarthritis. Patients in the trial receive two injections of MSCs into the affected joint within a few weeks, followed by a third dose one year later. The study includes a two-year follow-up to assess outcomes such as pain relief, joint function, cartilage structure, and overall quality of life. The final patient was treated at the end of last year, and the trial is expected to conclude in November.
Cynata has already completed two successful Phase 1 trials—one in GVHD, which led to an ongoing global Phase 2 trial, and another in diabetic foot ulcers. Pending the results of their current Phase 2 and Phase 3 trials, the company aims to validate the broad potential of MSC-based therapies.
Over the next five years, Cynata hopes to expand collaborations with both academic institutions and industry partners to bring the Cymerus platform to a wider range of diseases. Preclinical studies have shown promise for MSCs in conditions like idiopathic pulmonary fibrosis, where their anti-fibrotic effects may offer new treatment avenues.
With its scalable and scientifically rigorous approach, the Cymerus platform may play a pivotal role in advancing regenerative medicine and making MSC therapies accessible for many more patients worldwide.
By Impact Lab
