Category | Advanced therapy
Innovative gene therapies that utilize viral vector carriers such as adeno-associated virus (AAV) and lentivirus have the exciting potential to revolutionize today’s healthcare landscape by treating a myriad of previously untreatable and incurable genetic conditions, including rare cancers. Viral vectors are currently the most effective way to deliver genetic material into cells. They offer a groundbreaking avenue for addressing both inherited and acquired disorders at their root cause, deep in the human genome. By leveraging the natural ability of viruses to infect cells and insert their genetic material, scientists can specifically engineer viral vectors to transport corrective genes or modify existing ones within a patient’s cells. In fact, diseases like certain types of inherited blindness, blood disorders, and immune deficiencies have already seen promising results through innovative gene therapies that employ viral vector mechanisms. That said, the development and manufacturing of viral vectors is a complex and meticulous process for several reasons, and it requires sponsors to delicately balance considerations such as safety, efficacy, specificity, scalability, regulatory compliance, and stability, all while meeting the stringent quality standards required for clinical use. For that reason, many biopharma companies choose to partner with industry-leading and integrated CROs and CDMOs that have decades of experience developing and manufacturing viral vectors, rather than doing the work in-house.
A short clip from our recent webinar, “Benefits of an integrated approach to gene therapy development and manufacturing,” dives into the pros and cons of working with a third-party, integrated CRO/CDMO for viral vector development and manufacturing vs. taking a customized, in-house approach. The clip discusses five specific areas of focus, including 1) the number of service providers per product, 2) project timelines, 3) overall pricing, 4) performance and risk mitigation, and 5) necessary resources.
For the number of service providers per product, working with an integrated CRO/CDMO limits the number of vendors to one. In contrast, a customized model might mean working with six or more vendors simultaneously. Regarding project timelines, an integrated model can offer up to 16 months of savings, compared to a customized model that can potentially extend timelines. With pricing, an integrated model can provide an average of 20% savings when services are bundled. Although a customized model enables sponsors to shop for a lower price between vendors, it also incurs added costs for tech transfers. The clip also explores performance and risk management considerations, along with necessary resources. Regarding performance and risk mitigation, an integrated model ensures the same quality management system is utilized across all services, and it also gives sponsors access to a diverse pool of industry experts. Additionally, an integrated model enhances the ability to respond quickly to changes and it connects all critical deliverables. Conversely, a customized model allows sponsors to choose specific partners based on defined specifications, but that also requires sponsors to act as the liaison between vendors and coordinate alignment on key deliverables. Lastly, with necessary resources, an integrated model typically provides a dedicated project manager and single vendor qualification. Alternatively, a customized model requires the management of multiple vendors and varying processes.
Watch the webinar clip to gain a comprehensive understanding of the pros and cons of working with an integrated CRO/CDMO vs. taking an in-house approach. Additionally, explore the potential services and solutions biopharma companies would need at different phases of the viral vector development and manufacturing journey including during pre-clinical, phase I, phase II, and phase III clinical trials, Biologics License Application (BLA) approval, and finally commercialization.
To learn more about the benefits of an integrated approach for gene therapy development and manufacturing, watch the full webinar here.