Four Variables Impacting Cell And Gene Therapy Product Development Success

Robust and reproducible manufacturing processes are a critical differentiator in an increasingly competitive and crowded cell and gene therapy space. The FDA predicts that more than 200 applications will be filed per year from 2020 onwards, and 10–20 cell and gene therapy products will be approved annually by 2025. As more products make it to market, manufacturing processes are expected to improve as well. With a myriad of clinical trials currently ongoing worldwide, not every product is going to make it to the finishing line, let alone win the race to commercial success. Apart from clinical efficacy and safety, robustness and reproducibility of manufacturing processes are crucial to the ultimate success or failure of a product.

As more products are launched, some of the determinants of clinical and commercial success are being continuously re-defined. Obtaining regulatory approval for a product is a necessary but not guaranteed condition for success. Therapeutics developers with a first-to-market product, sound clinical data but poor manufacturing processes are in a fragile position to win the commercialization race. But one key success factor that will remain constant is the manufacturing process. As frequently quoted in the cell and gene therapy industry: “the product is the process and the process is the product”.

This article explores four key inter-connected variables exerting pressure on therapeutics developers to improve their processes earlier in the product’s development.