Viral Gene Therapy: Reducing Costs To Improve Patient Access

By Emmanuelle Cameau, Scientific Director of Viral Vectors, Cytiva

Gene therapy is rapidly emerging as one of the most transformative areas in the life sciences industry, offering groundbreaking potential to treat—and in some cases, cure—genetic disorders. The ability to precisely modify genes and deliver life-changing treatments to patients is nothing short of remarkable. In recent years, we've witnessed a steady rise in the number of approved cell and gene therapy drugs, with many more expected between 2025 and 2026. As the clinical trial pipeline continues to expand, it's conceivable that we could soon see over 25 new gene therapy approvals annually.

While the promise of these therapies is undeniably exciting, a critical issue remains: accessibility. Many of the most recent FDA-approved gene therapies, such as Roctavian®, Zynteglo®, Skysona®, and Hemgenix®, come with staggering price tags ranging from $2.5 to $3.5 million per dose. These costs have already led to regulatory and commercial setbacks, which include the withdrawal of Skysona from the EU market and Zynteglo’s exit from Germany due to pricing disputes with government officials.

As we look to the future of gene therapy, it's essential to not only celebrate the scientific advancements but also address the economic and ethical challenges that come with them. If we want these therapies to truly transform lives on a global scale, we must work toward making them accessible and affordable for all.