Innovative cell and gene therapy life science companies are poised to revolutionize disease treatment, significantly improve patient outcomes and transform the healthcare industry across a number of fronts, including patient access and treatment pricing models.
There are over 300 cell and gene therapies currently in clinical development targeting approximately 100 serious diseases (PhRMA). In 2017, Luxturna (Spark Therapeutics) was the very first gene therapy approved in the U.S. The past few years have seen several more treatments win FDA approval like YESCARTAⓇ (Kite Pharma) for non-Hodgkin lymphoma and KYMRIAHⓇ (Novartis) for large b-cell lymphoma, and, more recently, Novartis’s prospective gene therapy blockbuster drug ZOLGENSMAⓇ for spinal muscular atrophy.
This is just the tip of the cell and gene therapy iceberg.
By 2025, the FDA anticipates approving 10 to 20 gene and cell therapies every year (MSNBC). Clearly, cell and gene therapy will be an active disruptor to the global approach to human health in the very near future.
At Lab Owl, we don’t have to look very far to see how cell and gene therapies are disrupting the drug development and manufacturing processes. Right here in the BioHealth Capital Region (BHCR), which is rapidly emerging as a cell and gene therapy hub, American Gene Technologies, Kite Pharma, Autolus, REGENEXBIO, Rooster Bio, BeneVir Biopharm Inc., and Immunomic Therapeutics Inc. are each making significant advancements in the field.
The efficient development and manufacture of highly targeted, personalized cell and gene therapies requires a different approach than that of more traditional biologic upstream processes that are designed to produce large batches of material. Most cell and gene therapies are produced in much smaller batches. In particular, autologous (material taken from a single patient and returned to the same patient) cell and gene therapies are developed and manufactured for a single patient, taking what some call a “vein-to-vein” therapeutic approach.
This customized, targeted approach creates a unique set of challenges for cell and gene therapy companies when it comes to development and manufacturing. First, whereas traditional processes need to scale up, cell and gene therapies must find a way to scale out, or create multiple products simultaneously. Batch production is difficult to project, inventory management is challenging and supply chain logistics take on a retail-like model. Cell and gene therapy development requires open processing, often multiple, small, benchtop bioreactors, single-use manufacturing strategies, cutting-edge equipment and uberflexible manufacturing facilities.
This means more moving components, unpredictable production targets and coordinating and integrating complex upstream processes, including cell culture and fermentation.
In a recent GEN Q&A session about cell and gene therapy, Dr. Derek Adams, Chief Technology and Manufacturing Officer at bluebird bio, framed the paradigm shift this way: “In autologous cell therapies, the major pain point is that one cannot really scale up any unit operation…. The process is one patient, one batch—which limits batch volume and the associated economies of scale at that level. The small volumes and larger number of batches force replication of operations such as fluid movements, mixing, sampling, and labeling for every batch.”
Lab Owl, our automated bioreactor control and information system product, can play a crucial role for cell and gene therapy companies seeking greater control and process predictability.
CAR T-cell cultures, for example, are regularly conducted in bioreactors of less than 10L. This is a far cry from a few 2,000L bioreactors deployed for scale up in a more traditional upstream process. For autologous therapies, what this means is multiple, smaller bioreactors and benchtop set ups for custom product scale out to treat unique, individual patients at unpredictable manufacturing intervals.
A major cell culture and fermentation pain point is a lack of automation and poor bioreactor and information system control and integration. In other words, highly manual processes utilized by many labs are anathema to the customized, multi-dimensional and complex cell and gene therapy process.
Manual bioreactor control and information processes can no longer work in this environment.
We frequently encounter development labs struggling with a high level of manual process, which leads to inefficiencies, inconsistent results and siloed data. With so many moving parts, high material volatility and a stringent regulatory environment, lab automation, stronger bioreactor system integration and customization are imperatives for all cell and gene therapy companies.
Cell and gene therapy development and manufacturing is evolving as we speak. Best practices and GMP protocols are being developed and refined as more and more therapies move through the pipeline. Aaron Vernon, VP of Engineering and Supply Chain at Autolus Therapeutics, in his recent MAST 2019 keynote speech, likened the cell and gene therapy industry to the frontline of translational innovation, where processes and the rules of engagement are being learned on the fly and then standardized.
Lab Owl can help cell and gene therapy organizations stabilize and improve lab performance even in an industry that’s still trying to discover the best path forward.
Lab Owl is a technology agnostic, open platform, extremely flexible, bioreactor control and information system that is a technically advanced, modular and scalable solution for complex cell culture and fermentation processes. It is a solution that will not only stabilize lab performance at a reasonable cost, but is also able to adjust and scale as cell and gene therapy upstream processes develop over time.
Lab Owl is an alternative to one-size-fits-all, expensive automation products that are ill fit for the complexities of cell and gene therapy development and manufacturing. It is a remedy for the plague of resource-draining manual processes and it empowers development labs and growth-stage companies with a scalable, highly flexible and integrated bioreactor control system priced and built for their needs.
Precision medicine companies will radically transform the global approach to human health in the very near future. Lab Owl is proud to be part of the cell and gene therapy revolution that is unfolding right before our eyes, where established and startup organizations are pushing the boundaries of science to bring life-changing medicine to patients in need.