Category | Advanced therapy
The global gene therapy market is expanding at an unprecedented rate; some industry estimates predict it will reach over $38.76 billion by 2032, growing at a registered CAGR of 19.60% from 2023 to 20321. Its advancement is largely driven by industry demands for cutting-edge treatment modalities for a wide range of genetic conditions such as rare cancers, cystic fibrosis, and heart disease, among others. But to have their intended effect of altering genetic code, many gene therapies require viral vectors to effectively enter cells and introduce the new genetic material. Viral vectors derived from viruses like adeno-associated virus (AAV) or lentivirus are especially effective. Gene therapies utilizing viral vectors represent a stride towards increasingly personalized medicine. They have the potential to revolutionize how we approach and manage genetic diseases from the inside out. With that in mind, the development and manufacturing of viral vectors is a complex process with many considerations for biopharma companies.
From discovery to commercialization
To be approved for commercialization and widespread market use, viral vectors must move through several essential phases, including pre-clinical, phase I, phase II, and phase III clinical trials, and finally commercialization. For products in early phases, such as pre-clinical, phase I, and phase II, the primary focus for biopharma companies is largely on process development. For products in phase III, process characterization and robustness become the most critical. As products prepare for approval, biopharma companies must shift their attention to process performance qualification (PPQ), Biologics License Application (BLA) preparation, and continued process verification (CPV). Depending on which phase a product is in, be it pre-clinical, late-phase, or commercial, the main objectives for biopharma companies will also change. For instance, in the pre-clinical phase, discovery activities like proof of concept and lead identification are paramount. Phase I, II, and III all prioritize speed to clinic and robustness studies. In the final approval and commercialization phases, process control considerations rise to the forefront.
Depending on a sponsor’s strategy for viral vector manufacturing, and what development phase they’re in, they typically have the choice to insource key activities or outsource them to a third-party partner like a CDMO. When debating between a “make vs. buy” model, there are several areas for consideration including model objectives, project scope, site mechanics, manufacturing benefits, and financial factors. A short clip from our recent webinar, “Benefits of an integrated approach to gene therapy development and manufacturing,” specifically outlines the pros and cons of insourcing vs. outsourcing as they relate to every area for consideration. For example, for model objectives, insourcing allows for total control over the end-to-end manufacturing process whereas outsourcing enables biopharma companies to maximize savings while minimizing operating and utilization risks. Additionally, the insourcing model requires biopharma companies to construct a facility and hire employees from scratch, which can take several years. Conversely, outsourcing operations enables a fit-for-purpose approach, with ready-now capacity to immediately work on a range of viral vector projects.
Watch the webinar clip for a more detailed breakdown of each area for consideration and to understand better why biopharma companies choose to partner with third-party CDMOs to leverage their innovative, integrated, and ready-to-use solutions for viral vector development and manufacturing for gene therapies.
To learn more about the benefits of an integrated approach for gene therapy development and manufacturing, watch the full webinar here.