AABB2025: Biotherapies Field Undergoing Rapid Advancement as New Therapies “Come Across the Finish Line”

This year marked a significant achievement with the first mesenchymal stromal cell product approved by the Food and Drug Administration (FDA) for treating pediatric acute graft-versus-host disease—a milestone that was years in the making. In Sunday’s Cord Blood Connect President’s session, “Emerging Issues in Cord Blood Therapeutics and Gene Therapies,” Peter W. Marks, MD, PhD, noted that this is just one in a series of important recent advancements in cell and gene therapy, as well as regenerative medicine. Marks, the former director of the FDA's Center for Biologics Evaluation and Research, expressed optimism about the future of biotherapies, noting that the field is entering one of its most promising periods thanks to meaningful scientific milestones and an increasing number of clinical successes. 

“It’s a brighter time than it’s ever been before,” Marks said. “We’re finally starting to see cell and gene therapies come across the finish line.” 

Additional therapies are in development for a range of conditions, including muscular dystrophy and other genetic disorders. Although the pace of approvals has been slower than previously expected, both cellular and gene therapies are steadily advancing. Marks credited recent breakthroughs in genome editing and delivery systems, particularly innovations that emerged during the COVID-19 pandemic. 

Despite scientific advances, Marks acknowledged that regulatory uncertainty remains a persistent challenge in the United States. 

“We have to acknowledge the elephant in the room,” he told the audience. “There’s a lot of regulatory uncertainty right now. The output from FDA decisions is less consistent than it used to be, and it’s harder to predict which products will receive approval. As a result, some developers are turning to other countries where they can develop their products with more regulatory certainty, such as Australia, the United Kingdom or China.” 

Marks shared that when developers were asked what they valued most, regulatory certainty ranked higher than timely meetings with regulators. This data emphasizes the importance of clear, consistent guidance for innovators navigating complex approval pathways. 

Despite current uncertainties, Mark stated he is hopeful about the next chapter in regenerative medicine and encouraged by the tremendous progress in the field. He mentioned the FDA has expressed a commitment to supporting regenerative medicine and advanced therapeutic products. 

“When I was at the FDA, we were actively trying to develop a more risk-adapted way of regulating cellular therapies so that products were not all regulated either as simple tissues that don't require biologics licenses or full-blown biologics licenses,” Marks said. “It’s encouraging to see that discussion continuing today.”