Why validation is key to the industrialization of cell and gene therapies


*Corresponding author
1. BioPhorum, London, United Kingdom
2. Bayer U.S. LLC Pharmaceuticals, United States


A particular challenge associated with commercializing a cell and gene therapy (CGT) product is defining and performing a successful process and product validation campaign. This is particularly challenging due to limited experience, data and guidance currently available for CGT products. BioPhorum’s CGT validation workstream have published an article highlighting these issues, showcasing a number of unique validation challenges, and potential solutions, associated with CGT validation. Here we speak with one of the lead authors, Sharif Ahmed of Bayer, who kindly highlights some of these challenges, key takeaways/recommendations from the team, and the benefits of working as a cross-industry collaboration to discuss and identify potential solutions to common challenges.

Cell and gene therapy (CGT) products are a class of advanced therapeutics with tremendous potential in treating diseases. However, the challenges in manufacturing them are complex and industry is working to develop commercial capabilities, standard platforms, and sufficient capacities to address a growing pipeline. Successful commercialization of CGT products requires a multi-disciplinary approach that fully integrates patient needs and product knowledge with the capability to commercially manufacture these complex products consistently and reliably.


BioPhorum, a cross-industry collaboration organization, is exploring all aspects of CGT manufacturing and recently brought together a team of CGT and validation subject-matter experts from its member companies to discuss these challenges and advise industry through a white paper on ”Cell and gene therapy validation challenges” (1).


Beyond offering insights into the main CGT validation challenges, these experts make recommendations that may help companies devise strategies specific for their products in consultation with regulatory agencies. Product-specific strategies will be based o ...