Last week in Part 1 of our series, we presented observations from industry leaders into how they plan for success by installing comprehensive quality management systems and laying down detailed groundwork for an optimized transition to GMP-compliant starting materials and technologies. This week in Part 2 of our series, cell therapy manufacturers present their own perceptions of what constitutes an ideal cell therapy starting material supplier, as well as what can be done on their end to ensure their cell therapy retains consistent quality and potency.
The emergence of cell and gene therapies onto the global stage is generating growing excitement, as unprecedented clinical success fuels the expectation of a promising new chapter in medicine. Alongside an extraordinary increase in the number of cell and gene therapies entering clinical trial, there are a lower yet still significant number of companies launching the commercialized manufacture of their cell therapy products.
“As the industry and regulatory landscape continues to evolve, it’s vital that as an industry, we take a lifecycle approach to risk management –to ensure that we mitigate as much risk as possible all the way from discovery through to commercialization.” - Dr. Dominic Clarke, Global Head of Cell Therapy, HemaCare
Cell and Gene Therapy Insights has just published HemaCare’s white paper on how to manage starting material quality and stability to maximum effect during cell therapy manufacturing.  Quality cell therapies can only be created from quality starting materials, yet paradoxically, starting materials are the single greatest source of variability in the cell therapy manufacturing process. Mitigating risk requires minimizing that variability, and also managing shelf-life limitations and biopreservation logistics.
An independent publication in Nature: Scientific Reports cites using fresh leukopaks sourced from HemaCare in their T cell cryopreservation study. The authors, who work at the Cell and Gene Therapy Catapult in London, are studying the impact of cooling and thawing rates on cryopreserved human peripheral blood-derived T cells. In order for the emerging cell and gene therapy industry to effectively ship and store cell-based therapies to global markets, it will be necessary to cryopreserve cell therapy starting materials, as well as the final therapeutic products. To retain peak cellular function, and therefore therapeutic efficacy upon patient administration, it is necessary to understand how to optimize the cryopreservation process.
The U.S. Food and Drug Administration (FDA) has announced that they are adding staff and rolling out policy changes aimed at advancing the development of safe and effective cell and gene therapies.  The announcement came in the form of a press release on January 15th, citing that the new policies are a response to the current surge in cell and gene therapy products that the agency is handling. Based on the number of investigational new drug (IND) applications being submitted, the FDA projects a significant rise in the number of therapies that will be approved over the next few years: