Cell and gene therapy (CGT), once considered science fiction, is rapidly becoming a reality. From the success of CAR-T therapy in hematological malignancies to the approval of gene therapies for genetic diseases such as thalassemia and hemophilia A, the field has witnessed historic breakthroughs. In 2023-2024, several landmark therapies worldwide received approval from the US FDA, European EMA, and other agencies, heralding the arrival of an era of "one-time cures."
However, behind this explosive growth lie enormous challenges. First, there is the pricing and payment dilemma. The cost of a single treatment, often exceeding millions of dollars, places a heavy burden on health insurance systems and patients. This has spurred innovative payment models such as "pay-for-treatment" and installment payments, as well as innovations in insurance products. Second, there is the complexity of production and supply chain bottlenecks. CGT products are highly personalized, have long production processes, and require extremely high quality control; achieving large-scale, standardized production is a challenge facing the entire industry. Finally, there is the evolution of regulatory science. Global regulatory agencies are collaborating to establish more flexible review pathways to accelerate the accessibility of these breakthrough therapies to patients while ensuring their long-term safety and efficacy.
Looking ahead, trends will focus on the maturation of universal cell therapies and in vivo gene editing technologies, as well as addressing unmet clinical needs such as solid tumor treatment. Meanwhile, the maturity of the industry chain and cost control will become key to competition.
