How single-use technology will be critical to the commercialization of cell and gene therapies

Scaling up cell & gene therapies from research and development to commercialization is creating a new set of challenges for manufacturers. With the urgent demand of speeding up the market reach for these life-altering innovative therapies a high risk is inherent to implement inefficient workflow processes. This article will demonstrate, how adopting innovative single-use solutions for in-process sampling can have a tremendous effect in efficiency and effectiveness to the workflow.  Single-use solutions continue to provide productive strategies in effectively scaling up these processes while reducing risks and costs. 

Cell and gene therapies are an effective modality for treating and even curing complex diseases, with rapid advancements in therapy development that have resulted in significant regulatory approvals in recent years. The first CAR-T therapies, tisagenlecleucel (Kymriah® from Novartis) for pediatric acute lymphoblastic leukemia and onasemnogene abeparvovec-xioi (Zolgensma® from AveXis) for spinal muscular atrophy with children under the age of 2, are two examples of treatment and curative options that were not available with API drugs before. With more than 1,000 clinical trials in cell and gene therapy applications ongoing in this space, over the next few years a large number of these products are likely to come to market.

When compared to the production and delivery of monoclonal antibodies (mAb) therapies, moving novel cell and gene therapies products from process development and clinical stages to commercial, large scale manufacturing has introduced a host of new challenges. Bringing a new product to market will always present a multitude of challenges with logistics, infrastructure and finding highly skilled labor. Additionally, safety and efficacy of the therapy are always going to be a concern for the manufacturer, but the challenges facing these manufactures is resulting in new solutions having to be developed and designed to overcome these new obstacles to commercialization.

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Author

Timothy Korwan

Director – New Product Introduction, Single-use

Tim has more than 20 years of experience as an engineer and in business development with PAW BioScience Products, VWR and Biopure Corporation where he has designed single-use products, components and systems for the global drug and vaccine manufacturing industry.

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