Frost & Sullivan Released The 2026 Global Gene Therapy Industry Development Blue Book

Gene therapy represents a fundamental shift in disease treatment by addressing genetic abnormalities at their root, rather than merely managing symptoms. The report distinguishes gene replacement, which introduces functional gene copies for monogenic defects, from gene supplementation, which adds therapeutic genes to improve pathological mechanisms in more complex disorders. These approaches contrast with conventional treatments by aiming for long-lasting benefits, potentially with a single administration.

The evolution of gene therapy also includes gene expression regulation, which modulates endogenous gene activity without altering DNA sequences, and gene editing, which uses tools like CRISPR-Cas9 to make precise, permanent modifications to the genome. Gene editing is regarded as a therapeutic approach with the potential to deliver long-term benefits following a single treatment, by directly modifying disease-causing DNA sequences or regulating their expression.

As gene therapies advance through clinical development, the industry's competitive landscape is evolving. The report emphasizes that success is increasingly dependent on scalable manufacturing capabilities and robust Chemistry, Manufacturing and Controls (CMC). This indicates a move beyond pure research and development, where the ability to produce and control the quality of delivery vectors, such as viral and non-viral systems, becomes a critical factor for commercialization.