Overcoming the Next Frontier: Industry Experts Tackle Challenges in Cell and Gene Therapy at GenScript Biotech Forum 2025

The 2025 GenScript Biotech Global Forum brought together top minds in the biotech industry to reflect on key milestones and ongoing hurdles in the evolving field of cell and gene therapies (CGTs). The forum spotlighted the transformative potential of chimeric antigen receptor (CAR) T cell therapies, while emphasizing the need for strategic innovation to address lingering issues in manufacturing, scalability, distribution, and healthcare access.

CAR T cell therapy has come a long way since the foundational research began more than three decades ago. The first documented success occurred in 2010 with the treatment of a former Marine suffering from chronic leukemia. Two years later, Emily Whitehead became the first child to be cured using CAR T therapy, marking a turning point in the field. Since its formal FDA approval, nearly 50,000 patients globally—primarily those with blood cancers—have received this form of treatment, according to keynote speaker Dr. Carl June, director of the Center for Cellular Immunotherapies at the University of Pennsylvania.

Dr. June noted that 2024 represented a significant leap forward, with CAR T cell therapies expanding into glioblastoma and showing early promise in autoimmune disease management. Echoing this optimism, Alan Bash, president of Legend Biotech, affirmed that the therapeutic impact of CGTs is now clearly evident. “The products are delivering, and the products are demonstrating the innovation for patients,” he stated.

Yet despite these successes, critical challenges persist. A major theme across multiple panels was the gap in skilled labor and training. Michael Vreeland, US site head at ProBio, underscored the importance of workforce readiness, stating, “One of the things that’s often overlooked is the training piece… How do we ensure consistent and repeatable training and execution?”

Manufacturing and scalability remain complex and resource-intensive. Bash emphasized that while regulatory hurdles have diminished, the technical and logistical obstacles of production and distribution continue to strain the system. Rey Mali, chief business officer at Accellix, reinforced this point, noting that improvements in these areas are essential for broader patient access and cost control.

Jonathan Esensten, of Multiply Labs and Sheba Medical Center, added that reliance on manual processes in cleanroom environments still dominates current CGT manufacturing. While automation holds promise, Hari Pujar of Flagship Pioneering reminded attendees that individual patient cell variability still introduces a critical challenge to full standardization.

Another pressing concern is the limited capacity of treatment centers. Bash pointed out that the bottleneck now lies not just in manufacturing facilities, but in clinical sites themselves. Investors are increasingly focused on expanding CGT treatment capabilities beyond major academic hospitals into more accessible healthcare settings.

Supply chain fragility was also a recurring topic. Many vital CGT components currently depend on single suppliers, heightening the risk of delays. Additionally, the quality of patient-derived starting materials—often impacted by prior treatments—adds further complexity to therapy development. As Mali put it, “Your drug is only as good as what you start off with.”

Economic factors and investor confidence are additional concerns. Massimiliano Paganelli, CEO of Morphocell Technologies, noted that overinflated valuations in previous years have slowed investment, though a more mature understanding of CGTs is starting to reverse that trend.

International payer models present further difficulties. While the U.S. reimbursement structure poses challenges, panelists identified even greater complexity in regions such as the Asia-Pacific. Still, Louis Breton of Rampart Bioscience observed a growing focus on patient-reported outcomes, which could reshape how payers evaluate CGTs in the future.

Despite these obstacles, the industry remains optimistic. With venture capital shifting toward fewer but higher-value investments, and IPO numbers rising from six in 2023 to 18 in 2024, the momentum continues. Panelists agreed that automation and in vivo approaches—where therapeutic cells are generated directly within the patient’s body—could dramatically streamline the CGT process.

As Hari Pujar provocatively asked, “What if we could create the cell inside the body… in which case we kind of default all of the challenges that have been mentioned?” It’s a question that encapsulates both the progress made and the transformative potential still ahead.

Source: https://www.biospace.com/drug-development/beyond-the-breakthrough-industry-leaders-address-key-hurdles-facing-cell-and-gene-therapies

This is non-financial/medical advice and made using AI so could be wrong