Setting the Standard: Redefining Quality Control in Cell and Gene Therapy

Unlike traditional treatments, cell and gene therapies represent a paradigm shift in the therapeutics sector by targeting underlying cause rather than merely managing symptoms. These therapies modify patient’s genetic material or replace dysfunctional cells, offering curative potential for the treatment. With the rapid expansion of the cell and gene therapy pipeline in recent years, offers significant potential to treat diseases with great unmet medical need, highlighting the critical need for manufacturing quality control. Rey Mali, Vice President Sales and Marketing, Accellix, emphasizes “the limitless potential of CGT and the urgency of QC in meeting the growing demand for regenerative medicine, aligning with the article’s focus on scalable and efficient QC processes.”

Further, Jérôme Larghero, Director of the Department of Biotherapies and the MEARY Center for Cell and Gene Therapy in the Saint-Louis Hospital, AP-HP on the significant quality control in CGT manufacturing asserted that, “We all know that two healthy donors are different because their cells are different. At the end, we are supposed to have robust, reproducible processes, and much is based on how you will qualify your product and thus, which kind of quality control you will put in place to enter this drug into business and its reproducibility.”

Thus, the rapidly evolving landscape of clinical trials in cell and gene therapies globally is influencing how manufacturers approach quality control, with a range of strategic and operational changes in QC practices, as:

• The clinical pipeline for cell and gene therapies expands worldwide, manufacturers face heightened pressure to adopt scalable, standardized QC procedures that can efficiently manage with increasing volumes of products and provide batch-to-batch consistency.

• With growing clinical trials in many geographic areas, there is a growing need for international harmonization of QC requirements. Synchronization of analytical validation strategies and regulatory needs will be essential to enable smooth international product development and regulatory approval processes.

• The uneven geographic distribution of clinical activity highlights the need to build QC infrastructure in underrepresented regions. Local investment in QC capability, training and technology transfer will be essential to facilitating global trial diversity and equitable access to innovative therapies.

• In order to cope with trial complexity and trial volume, manufacturers are embracing automation, in-line analytics, and real-time monitoring of data. These technologies enhance QC precision, reduce manual errors, and accelerate product release timelines.

• The diversity of cell and gene therapies from live cell-based products to genetic modification requires different QC approaches. QC systems have to be specific to solve the specific analytical issues of each modality, e.g., cell viability tests for cell therapies and vector integrity tests for gene therapies.

Additionally, quality control in cell and gene therapy evolution is also aimed at critical need of a highly trained workforce capable of handling new technologies and following stringent standards. This concern was highlighted by well-known industry leader, Félix Alejandro Montero Julian, PhD, Scientific Director of the Healthcare Business of BioMérieux, “As the number of patients grows and the batches grow with them, this talent crunch is really going to threaten the growth of the industry.” In addition, Thomas Jones, Cell & Gene Therapy Business Senior Director for bioMérieux echoes Montero Julian’s concerns about the talent shortage in CGT QC and emphasizes the need for simplified QC tests to address high turnover and the lack of skilled personnel. “He highlights the rapid growth in patient numbers, citing projections like Kite’s goal to treat 25,000 patients annually by 2025 and the Alliance for Regenerative Medicine’s forecast of regulatory decisions on 17 new CGT products by 2023.” This underscores the urgency of scalable QC solutions to meet increasing demand. To overcome this, academic institutions and industry experts are working proactively to develop tailor-made training programs.The programs concentrate on hands-on certifications in GMP-compliant manufacturing, online quality check platforms, and cell and gene therapy specialized regulatory frameworks.

Peering into Tomorrow

As cell and gene therapies continue to redefine the boundaries of modern medicine, the quality control landscape must equally evolve in accordance to prioritize scalability, precision and compliance. The future of QC in manufacturing cell and gene therapy will be an integration of novel technologies such as digital twins, which support predictive modelling and real time optimization of manufacturing processes, and blockchain solutions that provide end to end transparency and traceability along the supply chain. This change will extend far beyond regulatory requirements to establishing new industry standards that protect consistency, reliability, and patient safety throughout each phase of the product life cycle.