SEPTEMBER - 20219TECH OUTLOOKproduction will require integrated, end-to-end systems incorporating a wider range of manufacturing expertise than with more traditional molecules.As with any new treatment, the regulatory landscape is fraught with uncertainty. Cutting-edge medical centers are seeing extraordinary success with engineered cell therapies modified by CRISPR achieving "cure" rates unimaginable just a few years ago; these therapies are, however, too expensive to scale. To effectively reduce the cost and broaden access to these treatments, manufacturers and drug developers must partner to define clear quality and regulator metrics to allow faster, safer production of the required substances that is scalable to the necessary level to meet patient demand. This need will become even more pronounced as cell and gene therapies shift from rare diseases to more common ones, such as oncology.Challenge 2: Mapping innovation in research to success in therapeuticsWith gene therapies, much more so than with the traditional drug development pipeline, researchers are closer than ever to the clinical relevance of their work. A robust, and ideally rapid, pipeline for translating innovation in new CRISPR tools, such as single base editing proteins, assures that the most suitable system is always being used rather than simply the most established one. These tools also rapidly expand the applicability of gene therapies that previously relied on DNA cleavage almost exclusively. Moving from the research bench to the clinic, however, presents a number of hurdles. Safely removing as many of these hurdles as possible will require suppliers, manufacturersand CDMOs that can more closely partner with, and provide materials to, scientists across the research-clinic spectrum.Solution: A focus on new modalities, from bench to bedsideClearly, the need for CRISPR-based therapies requiressubstantial scientific discovery and validation as it becomes more mainstream,shedding its label of `new' for a label of `proven'. Importantly, it will also require suppliers and manufacturers that can support the challenge of novelty and discovery, providing new tools quickly to researchers, as well as the challenge of manufacturing ready materials, including GMP-grade versions of the aforementioned tools that are appropriate for research through clinical development and validation. The ultimate success of gene therapies, and the patients they cure, will depend on the ability of both early discovery scientists and process manufacturing innovators to develop not just new technologies, but a new mindset for delivering these critical medicines to patients.
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