Navigating the Future of Advanced Therapies: Our Insights from Advanced Therapies Week07 Feb 2024
Advanced Therapies Week, Miami, recently showcased the dynamic future of advanced therapeutic development, with manufacturing and scalability taking centre stage. These critical areas are essential considerations if the industry is to meet the increasing global demand for advanced therapies as well as the utilization of these therapeutic modalities in larger disease indications.
Despite financial hurdles, the outlook for Cell and Gene Therapy (C>) is positive. The industry is seeking to deliver solutions targeted at areas of high unmet need in cancer and neurodegenerative diseases, among others. Although safety remains a top priority in therapeutic development, the industry is focused on boosting productivity to make these safe and effective advanced therapies more widely accessible and affordable. One potential approach that was discussed extensively is through the establishment of technology platforms. These highly optimized and chemically defined manufacturing processes utilize a “plug and play” feature to differentiate disease targets or patient populations with an anchor technology to facilitate clinical development. Indeed, with an expected rise in clinical evaluations and Biologics License Applications (BLAs), there was a consensus that regulatory processes must evolve to accommodate the unprecedented projected growth. Technology platforms are an attractive solution to provide facilitated regulatory approval timelines. However, even as the industry continues to evolve, the expansion of these advanced therapies from rare to more common diseases, scaling manufacturing and distribution are very likely to be the next significant challenges for the industry.
For Albumedix, now a part of Sartorius, the conference revealed varying levels of customer knowledge regarding albumin's function in biomanufacturing. However, there was a common concern with the use of plasma-derived albumin in manufacturing processes due to known inconsistencies present in this raw material. These inconsistencies have the potential to be incompatible with this novel platform approach through the introduction of variability in the process. Ensuring process consistency, which is crucial for the success of platformed technologies, hinges on the quality of raw materials and stringent manufacturing controls. Recombumin® recombinant human albumin stood out for its potential to improve various unit operations across the manufacturing continuum and deliver a consistent raw material input. Recombumin® is a reliable and high-quality tool for developers that supports process consistency and is an attractive alternative to plasma-derived albumin for platformed therapeutic approaches.
Advanced Therapies Week has affirmed the industry's resolve to tackle accessibility, safety, and regulatory hurdles. Undoubtedly, advanced therapies are set to transform the treatment landscape for debilitating diseases and offer the potential to change lives worldwide. Albumedix, now a part of Sartorius, is committed to supporting the industry in delivering on the promise of advanced therapies through its industry-leading albumin-enabled solutions.