The complexities of next-generation therapies are pushing the boundaries of development and manufacture, leading to an increased need for advanced technologies and novel approaches to processes.
45 cell and gene therapy (CGT) products approved by FDA
Novel therapeutic modalities, such as cell, gene, and nucleic acid therapies, are offering more effective, personalised treatment options to patients with chronic and/or rare diseases that were, not too long ago, considered to be untreatable. The latest regulatory data (at the time of writing) confirm that approvals of these therapies have generally increased year-over-year, with 45 cell and gene therapy (CGT) products approved by FDA and 27 advanced therapy medicinal products (eight of which have been withdrawn or not renewed) approved by EMA since the noughties (1,2).
However, these “next-generation” therapies, while extremely promising from a treatment perspective, are more complex than traditional drug products, meaning their development and manufacture are inherently more challenging. “Next-generation therapies, particularly those in the [CGT] space, are pushing the limits of what our current manufacturing infrastructure can support,” explains Edwin Stone, CEO, Cellular Origins. “These therapies are incredibly promising, but they’re also incredibly complex. You’re not just making a product; you’re engineering a living, and often patient-specific solution. That introduces variability, tight handling windows, and need for rigorous traceability.”
Complications
The inherently different scientific intricacies of next-generation therapies in comparison to traditional pharmaceuticals has given rise to challenging regulatory pathways, remarks Alexander Seyf, CEO Autolomous. “Demonstrating robust traceability, control, and consistency across the entire lifecycle, from raw material sourcing to final product release, presents a significant hurdle, particularly following manufacturing changes where maintaining product comparability adds further complexity,” he says. “Ensuring chain of identity and custody, upholding stringent quality and safety standards, and mitigating process variability are critical imperatives.”
Additionally, manufacturers are faced with scalability hurdles as a result of the complex nature of the therapies and limitations of currently available infrastructure, Seyf continues. “Many early-stage workflows, developed for academic or research settings, rely heavily on manual processes, hindering standardization and large-scale production. This results in quality inconsistencies, variable yields, and protracted timelines,” he says. “High GMP [good manufacturing practice] operational costs, scarcity of critical materials like viral vectors, and a shortage of skilled personnel further inflate expenses.”
Scalability is a common obstacle for bio/pharma organizations trying to manufacture next-generation therapies, confirms Stone. “Facilities designed for small-scale, manual processing simply don’t translate to commercial production. Labor is stretched, processes are fragmented, and regulatory risk increases. Despite great strides in getting the best out of the manual processes, the field is stuck trying to industrialize therapies using non-industrial tools,” he emphasizes. “There is a smarter way of achieving scale and that’s through automation for scale.”
How to by-pass limitations
With regard to next-generation therapies, companies are approaching drug development much more differently as compared with more traditional pharmaceuticals, notes Max Baumann, co-founder, partner, Treehill Partners. In addition, there are several technological advances available that can help companies overcome the challenges associated with the development and manufacture of these complex drugs.
References
FDA. Approved Cellular and Gene Therapy Products. FDA.gov (accessed May 22, 2025).
EMA. CAT Quarterly Highlights and Approved ATMPs. Committee Report, EMA.europa.eu, Feb. 10, 2025.