Mesenchymal stem cells (MSCs) have long shown promise in regenerative medicine, with the potential to treat everything from heart disease to autoimmune conditions. But for decades, MSC therapies struggled to move beyond early-phase clinical trials and regional approvals. That changed in December 2024 when the U.S. Food and Drug Administration (FDA) approved RYONCIL—and it became the first FDA-approved MSC therapy for pediatric steroid-refractory acute graft-versus-host disease (aGVHD). This landmark moment not only validated the therapeutic potential of MSCs, but also opened new possibilities for treating inflammatory and autoimmune conditions on a broader scale.
In our March 2025 edition of the Bridging the Gap webinar series—presented by Azenta Life Sciences and the Emily Whitehead Foundation—we were honored to explore the significance of this breakthrough with Dr. Anthony Ting, Chief Scientific Officer at Kiji Therapeutics and a long-time leader in cell and gene therapy. Dr. Ting was joined by our permanent panelist Albert Ribickas, Assistant Director of the Cell Therapy Facility at Moffitt Cancer Center, along with co-hosts Olga Bukatova from Azenta Life Sciences and Tom Whitehead from the Emily Whitehead Foundation.
From Milestone Approval to Future Momentum
Dr. Ting, who has worked in the cell and gene therapy space for more than two decades, emphasized how momentous the FDA’s approval of RYONCIL truly is.
RYONCIL, developed by Australian biotech company Mesoblast, treats a devastating complication of stem cell transplants—steroid-refractory acute GVHD in children. Until now, there were no FDA-approved MSC products for this indication. This approval, said Olga Bukatova, is “not just another regulatory win—it’s a turning point for our field.”
“It’s a milestone to finally have an approved MSC therapy in the United States. It’s been a very long journey,” said Dr. Ting. “Mesoblast had submitted their BLA several times and were denied, but they persevered. They strengthened their arguments and really made their case.”
Dr. Ting explained that the global MSC community, long collaborative and research-driven, is finally beginning to see the clinical fruits of decades of work. The FDA approval provides a critical framework for future MSC products to follow.
How We Got Here: A Shift in Understanding MSCs
Dr. Ting’s own journey in translational science began in academia and evolved through leadership roles at organizations like Takeda, Bone Therapeutics, and Athersys. Early in his career, the prevailing theory was that MSCs would work by differentiating into new cells. But over time, researchers discovered MSCs act more like “drug factories,” modulating immune responses and secreting therapeutic factors.
“That was probably a game-changer,” Dr. Ting explained. “We realized MSCs weren’t replacing tissue—they were interacting with the immune system to promote healing.”
This shift opened the door to broader applications in inflammatory, autoimmune, and degenerative diseases. It also prompted new manufacturing innovations, including efforts to scale up MSC production using microcarrier beads and bioreactors.
Next-Generation MSCs and What’s Coming Next
One of the most exciting developments in the field is the rise of gene-modified MSCs. Dr. Ting’s company, Kiji Therapeutics, is developing an engineered MSC product that expresses both CXCR4 and IL-10, aimed at improving homing to inflammation sites and enhancing regulatory T-cell activity.
“Just like in the CAR-T space, I think genetic engineering is going to make MSCs much more potent,” said Dr. Ting.
He also highlighted other promising technologies, including:
- Induced pluripotent stem cell (iPSC)-derived MSCs, which offer a more consistent and scalable supply.
- Companies like Healios (Japan) and Steminent Biotherapeutics (Taiwan) working on stroke, acute respiratory distress syndrome (ARDS), and neurodegenerative diseases.
- The emerging field of exosomes—MSC-derived vesicles that carry therapeutic molecules.
Dr. Ting noted exosomes are particularly exciting because they could one day offer a cell-free version of MSC therapy, potentially bypassing many of the challenges of cell-based manufacturing and regulation.
Expanding Access: Regulatory and Reimbursement Challenges
While the science has progressed, access remains a major barrier. As Tom Whitehead reminded the audience, “We have great technology, but we need to get it to more patients.” Dr. Ting echoed this sentiment:
“We need greater awareness among physicians and conversations with payers,” he said. “These therapies aren’t cheap, but for patients with no other options, the outcomes justify the cost.”
He called for better education across the board and pointed to the need for automation, AI-driven analytics, and standardized potency assays to reduce manufacturing costs and increase scalability.
Ribickas added that decentralized manufacturing platforms—like the Cocoon® and CliniMACS Prodigy® systems—are helping bring therapies closer to the bedside, improving both accessibility and standardization.
Looking Ahead: The Future of MSCs and Cell Therapy
As the field evolves, several trends are poised to shape the next five years:
- Gene-Modified MSCs: Increasing potency and expanding indications, including cancer.
- Exosome Therapeutics: A new frontier with regulatory and clinical potential.
- In Vivo CAR-T: A revolutionary approach that could eliminate the need for complex manufacturing.
- Improved Regulatory Pathways: There’s growing discussion around creating intermediate approval routes for less complex cell products.
- Patient Education and Advocacy: Continuing the work of the Emily Whitehead Foundation to ensure patient voices guide research and policy.
Reflecting on his career, Dr. Ting shared a personal story about delivering cells to patients via private jet in the early days of clinical trials—an experience that highlighted both the challenges and the urgency of translational research.
“When you’re there for the first patient receiving the therapy you developed—it changes you,” he said. “Every researcher should have that opportunity. It inspires you to work harder.”
Watch the Webinar
As always, Bridging the Gap remains committed to conversations that center on patient access, clinical innovation, and the transformative potential of cell and gene therapies. A big thank you to Dr. Anthony Ting for joining us this month and to all of our panelists and attendees for continuing to drive this field forward.
Be sure to register for our webinar series to receive updates, recaps, and invitations to upcoming webinar sessions.
About the Guest Speaker
Tony Ting, Ph.D.
CSO of Kiji Therapeutics
Dr. Ting is the CSO for Kiji Therapeutics, which is developing state-of-the-art off-the-shelf engineered cell therapies for multiple life-threatening diseases. He has over 30 years of academic and industry experience in translational science and global regulatory filing with over 20 years in the cell therapy field. He was recently the Chief Commercialization Officer for the International Society for Cell and Gene Therapy (ISCT) and now serves on the Cell Therapy Advisory group for the Alliance for Regenerative Medicine (ARM) as well as the Cell Therapy – Tracking, Circulation and Safety (CT-TRACS) committee for the Health and Environmental Sciences Institute (HESI). Prior to joining Kiji Therapeutics, he served as Program Leader in Oncology Cell Therapy Innovation at Takeda. Dr. Ting was the CSO for Bone Therapeutics, where he developed a novel induced-pluripotent stem cell platform for genetically engineered mesenchymal stem cells. Dr. Ting also served on the senior management team of Athersys as Vice President of Regenerative Medicine and Head of Cardiopulmonary Programs. He received his PhD from Johns Hopkins followed by a post-doctoral fellowship at Stanford.