In the burgeoning cell and gene therapy market, companies at all stages of pre-clinical and clinical development are faced with a similar set of challenges of producing effective, standardized, and compliant therapies. The novelty of these technologies combined with the rigorous quality controls surrounding the materials and production process require a specialized approach for starting material suppliers and therapy developers.
In a study of the European space, supply chain challenges were expressed by both large and small companies. (1) Certain large companies can set up their own testing and certification programs as a workaround for the shortage of suppliers who offer extensively qualified material, but this is not a viable option for most companies in this market. The result is a massive timeline and cost challenge for an industry which must use materials with a relatively short shelf-life produced by a small number of compliant suppliers.
This is a critical issue for the industry at large, given that the vast majority of therapeutic companies are still in the research or early clinical development stages, with expectations to grow their operations many times over. By the beginning of 2019, the FDA had only authorized 16 Advanced Therapy Medicinal Products (ATMPs) while the EMA had only authorized 13 ATMPs. (2) The American Society of Gene & Cell Therapy (ASGCT) provides a searchable database of 429 clinical trials for cell and gene therapy currently being conducted in the United States. 405 of these are still in phases 1 to 2.
The regulatory and logistical expansion required to go from the research stage to the clinical stage to full-scale commercial production is daunting. In an ideal world, companies creating novel therapies begin with the end goal in mind when developing their process, considering production at commercial volume. This might mean locking in dependable suppliers that offer materials with stage-appropriate quality at a cost that allows for discovery, testing, and industrial scale-up without notable changes to the production process and its associated documentation.
Akron is pushing to solve industry challenges from multiple angles. We are engaged in several efforts to standardize ancillary materials, for example, through the publication of our 3-stage approach to cGMP ancillary material development. (3) Furthermore, we are growing our manufacturing capacity to ensure security of supply for therapy developers looking to mitigate risks though long-term partnerships. This expansion will enable us to serve a greater aggregate demand with cGMP compliant ancillary materials. Akron’s quality compliance principles can be found here.
The industry is only just beginning to tap into the potential applications of these technologies. For example, in 2018, there were over 3,000 diseases listed on the NIH Genetic and Rare Diseases Information Center with zero FDA-approved drug therapies that are also thought to be treatable by gene therapy. (2) This is just one example of how much the future holds for the cell and gene therapy industry. If we hope to realize the promise of these technologies, the industry must focus on expanding the ancillary material supply chain. At the same time, suppliers have a responsibility to work closely with therapy developers to ensure the most cost-effective and sustainable solutions are being applied in each stage of the development process.
This is exactly what we do here at Akron. With our partners at Arcline, we will be expanding our manufacturing capacity and broadening our existing set of capabilities to drive growth and quality into the ancillary material supply chain, pushing this industry towards the destination that everyone hopes it will reach in the near future.
- Ham, R.M.T., Hoekman, J., Hovels, A.M., Broekmans, A.W., Leufkens, H.G.M., & Klungel, O.H. 2018. Challenges in Advanced Therapy Medicinal Product Development: A Survey among Companies in Europe. Molecular Therapy Methods & Clinical Development. 11(14):121-130.
- Seoane-Vazquez, E., Shukla, V., Rodriguez-Monguio, R. 2019. Innovation and competition in advanced therapy medicinal products. EMBO Mol Med. 11(3):e9992.
- Ball, O., Zylberberg, C. 2019. Towards a common framework for defining ancillary quality across the development spectrum. Cytotherapy. 21(12):1234-1245.