By Emmanuelle Cameau, Strategic Technology Partnership Leader - Cell and Gene Therapy, Pall Corporation
Originally published in Pharma Focus America, Issue 1
Gene therapy is a fast-growing sector in the life sciences industry, and there are countless reasons to be enthusiastic about it. The fact that we can use specific gene-modifying technology to amend genetic disorders and provide patients with such impactful treatment is amazing. We have seen an increase in drugs approved over 2021 and 2022, and there are several others pending approval in 2023 - 2024.
When we look at the clinical trial pipeline, one can only wonder whether we could reach 10 to 25 approvals per year by 2025. The idea of having all these approved potentially curative therapies on the market is certainly exciting. However, the question of the accessibility to these drugs by the wider population is still unanswered. Indeed, the last 4 drugs approved by health authorities range between 2.5 and 3.5 million dollars per dose. In some cases, regulatory agencies have even withdrawn approval as a direct result of the pricing.
Gene therapies for ultra-rare diseases will be commercially viable only if we drive costs down. There is a need for real innovation to help shift the paradigm of the cost per dose. Learn from an industry expert how a combination of process intensification and optimizing your process development can help drive the cost of dose down.