CAR-T Therapy Enters a Golden Era: Breakthrough Efficacy, Global Expansion, and Payment Innovation

Recently, the CAR-T cell therapy field in China has seen a series of milestone breakthroughs in clinical efficacy, indication expansion, international progress, and key payment stages, marking the transition from technological maturity to a new phase of industrialization and improved accessibility.

On November 3, CARsgen Therapeutics announced the clinical data of its universal BCMA CAR-T product CT0596 for the treatment of relapsed/refractory multiple myeloma (R/R MM), as well as the clinical data of its universal CD19/CD20 CAR-T product CT1190B for the treatment of relapsed/refractory non-Hodgkin's lymphoma (R/R NHL). The data showed that both universal CAR-T products demonstrated preliminary good safety and encouraging efficacy signals.

More strikingly, the application boundaries of CAR-T therapy are extending from cancer to broader disease areas. A research team from Tongji Medical College of Huazhong University of Science and Technology published a study in the journal Cell showing that the use of IASO Bio's anti-BCMA CAR-T therapy for Progressive Multifocal Sclerosis (PMS) achieved safe and effective preliminary results. This opens up a whole new avenue for the application of CAR-T technology in autoimmune diseases, demonstrating its tremendous therapeutic potential.

As ex vivo CAR-T therapy has exposed challenges in commercial applications, such as complex preparation processes, high production costs, susceptibility to cell function damage, and limited patient accessibility, in vivo CAR-T technology is rapidly emerging as a core direction for next-generation cell therapies. In vivo CAR-T technology uses delivery systems like LNP-mRNA to directly inject genes encoding CAR into the patient’s body, utilizing the body's own cells to generate CAR-T cells in vivo. This technology aims to transform CAR-T therapy from an expensive "personalized customization" treatment into an "instant, mass-producible" common drug, with the potential to fundamentally resolve current bottlenecks in CAR-T therapy, such as long production cycles and high costs.

International pharmaceutical companies have accelerated their deployment in this field through multiple high-value acquisitions. Since 2025, giants such as AstraZeneca, AbbVie, Gilead, and BMS have bet on in vivo programming platforms via acquisitions. In China, companies like Everest Medicines, DeepBiome, PersonGen BioTherapeutics, and Grail Bio have all entered related fields. Although most are still in preclinical or early research stages, they are rapidly catching up and participating in global competition.

While clinical data shows impressive results, the globalization pace of China-produced CAR-T has upgraded from regional layout to global rights collaboration. On October 21, IASO Bio announced that its BCMA CAR-T product received implied permission for clinical trials in Japan, officially opening the door to the East Asian market. Just one week later, the company signed a cooperation agreement with Korea’s GCCell to push forward the entry of Fokas® into the Korean market, initiating the registration and commercialization process, and building a clear blueprint for the Asia-Pacific layout.

IASO Bio Fokasol (Idecabtagene Vicleucel Injection)

For a long time, the high cost of treatment has been an insurmountable barrier between patients and CAR-T therapy. Over the past four years, all CAR-T products have failed in the national medical insurance negotiations due to pricing issues.

On November 2, significant news came from the national medical insurance negotiation site: HyCyte "has successfully negotiated." This means that Naciorl Injection, priced at 999,000 yuan, has completed price negotiations for the first time through the newly established "Commercial Insurance Innovative Drug List" and is expected to enter the medical insurance system, paving a new way for high-priced innovative drugs to gain market access.

Source: Insight Database

According to media reports, the CAR-T products from IASO Bio and CARsgen Therapeutics participated in this year's commercial insurance directory price negotiation. After the inclusion of Naciorlase at a price of 999,000 yuan per dose, the annual treatment cost for patients is expected to drop significantly thanks to reimbursements from various supplementary insurances. This breakthrough clears the final hurdle in payment for the entire innovative drug industry, with market expansion on the horizon.

CAR-T TherapyContinuous breakthroughs in clinical, commercial, and payment sectors indicate its vast application prospects and industrial potential. The vigorous development of the industry is inseparable from the robust R&D support system behind it. As a crucial link in the drug R&D industry chain, MedChem Express (MCE) is playing an indispensable enabling role.

MediClarity, as one of the few one-stop preclinical R&D service platforms for biopharmaceuticals in China, has established a mature cell and gene therapy drug R&D service platform. It covers various immunotherapy approaches including CAR-T, TCR-T, CAR-NK, and TIL cells. Utilizing a wide range of animal models and multiple advanced analytical technologies, and taking into account the characteristics of different research projects, MediClarity has successfully completed preclinical development projects for several immunotherapy solutions for its clients. It has effectively contributed to the approval of seven cell therapy drugs for clinical trials, accumulating solid project experience and technical expertise.

With the enhancement of efficacy, improvement in payment systems, and the advancement of globalization, the market size of CAR-T in China is expected to exceed 50 billion yuan by 2030. Technological breakthroughs in solid tumor research, universal CAR-T, and in vivo CAR-T are expected to extend coverage to millions of patients with gastric cancer, liver cancer, and more, unlocking a market potential worth hundreds of billions. The MDCyte Cell and Gene Therapy R&D Service Platform will continue to support the development of more CAR-T drugs, jointly promoting the progress of the global cell therapy industry.