SCALABLE, HIGH PERFORMANCE SINGLE-USE TECHNOLOGY TO MEET GENE THERAPY PRODUCTION DEMANDS

摘要

The biotech world is currently living through an exciting time of discoveries, making use of gene-base therapies for the treatment of diseases that previously had no cure. There is currently a large number of gene therapies in both R&D and clinical stages, and it is expected that demand for technologies able to address the industrial manufacture of these will boom in the coming years and decades. The main challenges associated with scaling up gene therapies from their currently R&D and clinical stage to the market will be to ensure robust and scalable technology is available through the complete process. Gene therapies typically require high doses per patient, while the patient pool size on average is much smaller than for traditional medicines. As a result, technology needs to be adapted to produce such high titers at reasonable costs. Additionally, technology also needs to be flexible enough to accommodate a range of therapies and vectors with ease. Univercells will present a case study on how it has developed single-use cell culture technology to address the challenges cited here above, and how it has as a result improved reproducibility, reduced both capital and operating costs and removed the scalability bottleneck between clinical trials and full commercial production.