Aside from the clinical opportunity that gene therapy products present; they do introduce tremendous complexity into both the manufacturing and commercial processes. Remember these three points before developing your gene therapy plan.
Looking back at the hurricane season from the perspective of supply chain risk — and specifically risks that have impacted the life sciences supply chain — there are many lessons to be learned.
Hear insights from Sam Herbert, president of World Courier, regarding how the evolution of the supply chain in traditional small and large molecules is a handoff from clinical partners into commercial partners.
Cell and Gene Therapies have demonstrated real promise as they move into the clinical trial phase for a variety of life changing and deadly diseases. The results of these studies and their potential impact to patients, their loved ones and the industry as a whole are almost incalculable, which makes securing the supply chain mission critical — though not easily accomplished. What makes World Courier so essential for Cell and Gene Therapy logistics is our understanding and our ability to address these critical issues.
Cell therapies have the potential to revolutionize the biopharmaceutical world, but today’s processes, logistics, and delivery make for a challenging entry into the sector’s growth curve. As the industry evolves, we have to answer (at least) three important questions when bringing these exciting new therapies to market.
In preceding articles in this series, we reviewed some of the challenges and remediation approaches for the storage and distribution of life science products, highlighting some of the specific risks related to storage, transportation, and material control across an extended chain of custody. This brings us to the final article, in which we will discuss best practices for selecting the partners that will be an extension of your staff for monitoring and control across an ever-changing global landscape.
With the number of biologics now in development and soon to be making their way into clinical trials, the preservation of cells, tissues, and organs are suddenly increasing in importance. Companies handling the manufacturing, storage, and transportation of these materials need to be focused on improving the yield and extending the shelf life of these time and temperature sensitive biologics.
The recent FDA approval of Sarepta’s eteplirsen (Exondys 51), while generally welcomed in the Duchenne muscular dystrophy (DMD) community, was not without controversy. Eteplirsen is a gene therapy drug for use in DMD patients whose dystrophin mutation is amenable to exon 51 skipping. It was approved after a study of 12 patients, four of whom received placebo for 24 weeks before being re-randomized to one of two dosages. Questions have arisen regarding the use of historical data as a control in the absence of a concurrent placebo arm of the latter part of the study, the efficacy measures used, and the use of post-hoc analyses conducted by the study sponsors. But in the end, Eteplirsen was approved by the FDA, and the process, regardless of attendant controversy, illustrated the potential for innovation in clinical trial design and methods of analysis.
The complexity associated with manufacturing a ‘living drug’ should not be underestimated and it is important to consider that each product will have its own specific challenges and complexities.
Distributing sensitive biopharmaceuticals and cell therapies is a complex challenge that calls for detailed understanding of the product, latest technologies, and a global reach.