How Cynata Therapeutics is Unlocking the Power of iPSCs to Transform Regenerative Medicine
By Erin Harris, Editor-In-Chief, Cell & Gene
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Cynata Therapeutics, a Melbourne, Australia-based regenerative medicine company, is pioneering a new era in cell therapy with its Cymerus platform. Built on the foundation of induced pluripotent stem cell (iPSC) technology, Cynata’s approach offers a transformative alternative to traditional mesenchymal stem cell (MSC) therapies, promising consistency, potency, and scalability that could redefine how cell therapies are manufactured and delivered.
At the heart of Cynata’s innovation is Cymerus, a manufacturing platform capable of generating a near limitless supply of MSCs from a single donor-derived iPSC line. This strategy addresses two longstanding issues in cell therapy: the inter-donor variability that affects product consistency, and the loss of cell potency due to repeated culture expansion. By scaling at the iPSC stage, before MSC differentiation, Cymerus sidesteps these pitfalls, producing a uniform product on an industrial scale.
“Our platform directly addresses both issues,” explains Dr. Kilian Kelly, CEO and Managing Director at Cynata. “First, as we avoid the need for new donors, we do not have to contend with variability between donors. Second, since we get our scale at the iPSC stage, we avoid extensive culturing after MSCs are formed.”
Promising Clinical Progress
Cynata has already achieved significant milestones with its iPSC-derived MSCs. In a Phase 1 trial for acute graft versus host disease (aGvHD), their lead candidate CYP-001 demonstrated notable results. Of the 15 patients with steroid-resistant aGvHD, an area with limited treatment options, 87% showed an overall response, and 53% achieved complete response. Indeed, the two-year survival rate hit 60%, an improvement compared to historical rates below 20%.
“This trial was the first completed clinical study worldwide using iPSC-derived cells in any disease,” says Dr. Kelly. “It generated highly encouraging response and survival rates, with no safety concerns — a key differentiator from current treatments like ruxolitinib, which, while approved, hasn’t shown long-term survival benefits and carries a problematic safety profile.”
Cynata’s second completed Phase 1 trial evaluated CYP-006TK, a topical MSC-seeded wound dressing for diabetic foot ulcers (DFUs). The product achieved up to 84% wound surface area reduction at 24 weeks in the treatment group, which was nearly double the reduction seen in controls.
With Phase 2 and Phase 3 trials now underway for aGvHD, kidney transplantation, and osteoarthritis, the company is on track to deliver critical data over the next year, including the Phase 3 results for CYP-004 in osteoarthritis expected in early 2026.
A Potential Disease-Modifying Therapy for Osteoarthritis
Cynata’s CYP-004 program in knee osteoarthritis represents a particularly exciting opportunity. Currently, there are no disease-modifying therapies approved for osteoarthritis, as only symptom management options exist.
“If successful, CYP-004 could become the first approved disease-modifying agent for osteoarthritis, which would be transformative for both Cynata and for people with this condition,” Dr. Kelly says. “Delaying or avoiding the need for knee replacement surgery would offer better quality-of-life and economic benefits.”
Administered via intra-articular injection, CYP-004 targets not only inflammation but the degenerative mechanisms underlying joint deterioration. With the final patient follow-up for the Phase 3 study anticipated later this year, the program is entering a critical phase.
Differentiation Through Secretome Profile and Engineering Potential
Cynata’s MSCs have shown consistent therapeutic effects across numerous preclinical studies, including respiratory, cardiovascular, and immunological models. One factor driving their effectiveness may be the MSC secretome, a complex mixture of proteins and bioactive molecules believed to mediate much of the cells’ therapeutic action.
A recent study compared the secretomes of various MSC sources and found that Cymerus MSCs featured more unique proteins and stronger immunomodulatory effects. In wound healing assays, iPSC-derived and umbilical cord-derived MSCs outperformed those from bone marrow and adipose tissue.
Cynata’s iPSC platform also opens the door to next-generation applications such as engineered MSCs for oncology. In this approach, MSCs act as delivery vehicles for targeted anti-tumor agents, leveraging their natural homing ability to infiltrate tumors.
“Our platform allows for engineering at the iPSC level, followed by consistent and scalable MSC production,” Dr. Kelly explains. “That’s a major advantage over donor-derived MSCs, where every new donation would require re-engineering, which adds cost, complexity, and variability.”
Cynata’s Future Directions and Commercial Strategy
While Cynata sees promise in a broad range of conditions, including pulmonary fibrosis, asthma, ischemic heart disease, and sepsis, it remains focused on advancing its lead programs toward commercialization. Manufacturing, a key challenge for many cell therapy developers, has been streamlined through partnerships with CDMOs, including Fujifilm.
“We’ve worked with CDMOs since day one and believe this model allows us to scale efficiently while controlling costs,” Dr. Kelly says. “Operating our own facility isn’t on the immediate horizon.”
A New Standard for Cell Therapy?
With its proprietary platform, encouraging clinical results, and a differentiated approach to cell therapy manufacturing, Cynata is positioning itself at the forefront of regenerative medicine. If Cynata’s iPSC-derived MSC therapies continue to deliver, they may well prove that consistency, scalability, and potency are not mutually exclusive in regenerative medicine — they’re the future.