Cell and gene therapy (CGT) products are a class of advanced therapeutics that have tremendous potential to treat diseases. However, their manufacturing challenges are complex and successful commercialization of CGT products requires a multidisciplinary approach that integrates patient needs and product knowledge with the capability to commercially manufacture these complex products consistently and reliably.
The basic principles of current good manufacturing practices and general process validation concepts and practices apply to CGT manufacturing processes and analytical methods; however, following validation, concepts established for general biologics can result in challenges for CGT product validation due to their unique characteristics. Therefore, CGT validation requirements are still in development. Specific challenges in validating CGT product manufacturing processes include identifying quality attributes, establishing specifications, and validating analytical methods.
This commentary highlights the published work from the BioPhorum Validation workstream indicating the unique challenges and possible solutions when attempting to validate cell and gene therapy products.