Global Gene Therapy Industry Development: From Gene Therapy Innovation to Industrialization and Commercialization

Follow the evolution of gene therapy from early genetic intervention to increasingly diversified therapeutic strategies. The Blue Book examines four major approaches — gene replacement, gene supplementation, gene expression regulation, and gene editing — together with the development of related delivery technologies and their role in expanding the application of gene therapy.

Examine the development of the global and Chinese gene therapy markets, including market size, industry structure, key development drivers, market opportunities, and the changing competitive landscape. According to the Blue Book, the global gene therapy drug market is projected to grow from approximately US$0.1 billion in 2021 to US$32.0 billion by 2035, while the Chinese market is expected to reach approximately RMB22.8 billion by 2035. The global market is expected to move from an early high-growth stage, driven by product approvals, accelerating clinical translation, and a low base, toward a more stable expansion phase as the industry progresses from early commercialization to broader-scale application.

Explore the production process of gene therapy products, covering upstream cell culture and vector production, transfection or infection, downstream purification, potency testing, impurity control, and scale-up. Particular attention is given to manufacturing challenges such as production yield, empty capsid rates, purification efficiency, production costs, batch consistency, and the ability to move from laboratory-scale production toward industrialized manufacturing.

Review the clinical development and application of gene therapy across major therapeutic areas, including ophthalmic diseases, genetic hematological disorders, rare diseases, central nervous system diseases, oncology, cardiovascular diseases, and metabolic diseases. The Blue Book also examines the progression from clinical development to commercialization, including treatment value, cost considerations, and payment approaches such as commercial health insurance, installment payments, and risk-sharing mechanisms.

Examine how regulatory policies, capital investment, industry collaboration, and cross-border development contribute to the evolution of the gene therapy ecosystem. The Blue Book also reviews representative gene therapy companies, their technology platforms, product pipelines, clinical progress, and development strategies, providing a view of how R&D, manufacturing, clinical development, and commercialization capabilities are being integrated across the industry.