One of the overarching themes of the Phacilitate conference this year was bringing down the cost of goods so cell and gene therapy (CGT) treatments will be more affordable. While “affordability” is relative in that the price for CGT’s, which is typically a single treatment, must be compared to a lifetime of enzyme replacement or cancer treatment, there is no doubt we still have plenty of opportunity to streamline the manufacturing and testing processes and reduce development costs. In order to realize these efficiencies, innovation is needed at all stages, from R&D developing less expensive delivery systems and more predictive analytical tools, to CDMO’s establishing GMP-grade supply chains and scale-up capabilities for complex biological products.
In recent years, clinical and commercialization strategies for CGT’s have been overriding extensive innovation at earlier stages in an effort to bring life-altering treatments to patients faster. Part of the challenge is that the treatments and delivery systems are so specialized that a platform approach for manufacturing and testing is not yet feasible. For cell therapies in particular, the manufacturing process is so closely tied to the product itself that it cannot be easily scaled up or transferred to multiple sites. With regards to gene therapy, the structure-function relationship for viruses is more complex than traditional biological proteins, requiring a new paradigm for analytical techniques. Lack of available reference standards and data management systems make streamlining analytics for CGT products challenging.
Overall, these challenges are good for the industry because it means the therapies are working. Now that we have seen commercial success with the likes of Kymriah, Yescarta, and Luxturna, we must learn from other established therapeutic modalities, like monoclonal antibodies and pharmaceuticals, to improve efficiencies with regards to supply chain, automation, manufacturing, and standards.
Written By: Karen Doucette, MBA, Director of Operations, ACF Bioservices
ACF Bioservices (an Absorption Systems’ Company) has the expertise and relevant experience needed to provide analytical support for gene and cell therapy products through every stage of development. Potency assays are on the critical path and ACF offers de novo development, optimization, qualification, validation and GMP compliant final product and product release testing services for both allogenic and autologous therapies.
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