Progress made in hope manufacturing

Hope is, in and of itself, a cure; imagine giving a true hope to treat a disease!

Humanity is witnessing progression at a pace like never before. This acceleration in progress have touched almost every sector in our life, including the cell and gene therapy field.

For any progress to meaningful, it has to involve every ring of the supply chain. We constantly see positive outcomes in cell and gene therapy because progress is made on all fronts:

• Research: Building on current and previous breakthroughs, researchers continue to utilize the knowledge to design materials and equipment that better serve the intended applications. These innovations feedback in research as seeds to result in more innovations, and the cycle continues.
• Materials: With the advancing technology in biology, chemistry, and physics, manufacturers collaborate with researchers to design reagents with applications and quality so each can fit specific research goal. They also collaborate to design material that aid in cell-based production by elevating both the quality and quantity of the cellular product.
• Equipment: Utilizing the innovative research and quality material, manufacturers built systems that are able to take the production of cell-based products to the good manufacturing practice level. A step that is widely thought of as enhancement to production by standardizing the process and eliminating man-made errors.
• Workforce: Personnel with expertise in cell and gene therapy are needed to do the complex processes. Majority of experienced staff get trained in production facilities with research and development capacity. Availability of these expertise does not meet the demand yet, but is systematically being increased via the dedicated capacity building programs.
• Platforms: Production platforms are still considered the bottle neck of the cell the gene therapy production due to the associated cost. However, progress has been made here too, and we now have alternatives that could be delivered in only potions of the usual cost and time.

It has been more than 40 years since the first successful bone marrow transplant in 1979, we are now in a totally different arena. The pace at which cell-based innovations are made is fueled by the remarkable supply chain. This pace has accelerated even more in the last two decades and will continue in the next decade and beyond.

• Clinical Trials: Successful translational research starts at the clinic as a clinical trial. celltrials.org data indicates that the number of the registered cell-based clinical trials has jumped from ~300 in 2011 to ~930 in 2020. Additionally, the number of registered immunotherapy trials increased from ~10 in 2011 to 290 in 2020. This drastic increase in clinical trials reflects the upstream progress that resulted in a full cell-based innovation pipeline.
• Approvals: Regulatory approval of therapeutic candidates indicates the successful completion of the clinical testing at all phases. The approval makes the therapeutic product accessible by a larger population of patients. Approvals of cell -based products by the food and drug administration (FDA) has accelerated over the last decade. With the dendritic cells being the first FDA approved cellular product in 2010, the list of approved products grown to 23 products by the beginning of 2022. The chimeric antigen receptor (CAR) T cells alone gained 5 approvals between 2017 and 2022; jumping from 1 to 5 in just 5 years. Furthermore, in a joint statement by FDA’s commissioner and director of the center for biologics evaluation and research, they expected the FDA to approve 10-20 cell-based products until 2025. The European medicines agency (EMA) approval pace has a trajectory similar to that of the FDA. Based on the submission of marketing authorization, pharma consultants expected the EMA approval to pick up in 2021. The EMA had approved their 1st advanced therapy medicinal product (ATMP) in 2009, by end of September 2021 the approved ATMP products have grown to 17.

A review of the cell and gene therapy landscape indicates that products of higher quality and lower price tags are on their way to approval. This generates excitement to cell therapy professionals and enables them to treat their patients with even more confidence. This review also provide hope to patients as many of their disease are now no longer considered untreatable.

This excitement and hope require more work upfront to ready the local supply chain, the platforms, and the approval systems. Bringing these systems to readiness is time consuming, therefore, should be carefully planned and integrated into the transformation activities of the health care system.

Let us know if we missed anything, or if you would like a specific subject to be discussed, write to us. 

About the author

Naseem Almezel, earned his MSc degree in Cellular Therapies in 2010, since then his career focus is to support Bone Marrow Transplant and Oncology programs. Naseem likes to work in the lab doing translational research, or in the cleanroom doing GMP production. When he is not working, Naseem likes to read and to spend time outdoors. Find more about Naseem here