China Leads Global Cell and Gene Therapy Trials with Over 50% Share, Showcasing Innovative Pipeline

　　【Pharmaceutical Network Industry Dynamics】In the wave of iterative upgrades in medical technology, cell and gene therapy have become crucial directions for tackling difficult-to-treat conditions such as cancer and genetic diseases. Against the backdrop of rapid global development in cell and gene therapy, Chinese scientists are contributing Chinese wisdom to the progress of world medicine through innovative technologies and clinical practices. According to information released at the 2025 China Cell and Gene Therapy Conference, there are approximately 2000 cell and gene therapy clinical trials globally, with China accounting for over 50%.
 

Analysis points out that in recent years, China's cell and gene therapy field has achieved leapfrog development by leveraging the multiple advantages of policy support, scientific research investment, and industrial collaboration. At the policy level, China has successively introduced policies that explicitly include cell and gene therapy as key development areas, optimizing the review and approval processes to clear obstacles for technological innovation and achievement transformation. In terms of scientific research investment, the country has continued to increase financial support for this field, promoting the deep integration of basic research and clinical applications.
 

Moreover, breakthroughs in innovative technologies are the core engine driving the rapid rise of China's cell and gene therapy field. It is reported that pharmaceutical companies in China have been making continuous progress in key technical areas such as CAR-T cell therapy, gene editing, and stem cell therapy, achieving a series of accomplishments.
 

On December 29, 2025, CARsgen Therapeutics announced that it had submitted two Investigational New Drug (IND) applications to the China National Medical Products Administration (NMPA) for its off-the-shelf BCMA CAR-T product CT0596, aiming to initiate Phase 1b/2 clinical trials for the treatment of relapsed/refractory multiple myeloma (R/R MM) and primary plasma cell leukemia (pPCL), respectively. CT0596 is an off-the-shelf CAR-T cell therapy targeting BCMA, developed based on CARsgen's proprietary THANK-u Plus® platform. By knocking out the NKG2A, TRAC, and B2M genes, it reduces the risk of graft-versus-host disease (GvHD) and host immune rejection, with additional gene editing further blocking host NK cell-mediated rejection responses, thereby enhancing the product’s efficacy and safety. CT0596 has already been under investigator-initiated trials (IIT) in China, exploring its clinical potential in treating R/R MM and pPCL.
 

On December 24, 2025, iRegene announced that its self-developed NouvNeu001 injection had been granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA on December 22. In August this year, the FDA also granted the drug Fast Track Designation (FTD) for the treatment of Parkinson's disease. According to reports, iRegene's NouvNeu001 (human dopaminergic progenitor cell injection) is a chemically induced functional human dopaminergic neuron progenitor cell. It is a clinical-stage, chemically induced, off-the-shelf iPSC (induced pluripotent stem cell)-derived cell therapy product. The product is derived from umbilical cord blood and uses human-induced pluripotent stem cells (iPSC), which are differentiated through a combination of chemical small molecules to obtain partially matured dopaminergic progenitor cells with the ability to differentiate into dopaminergic neurons after transplantation in vivo.
 

In January 2025, the Investigational New Drug (IND) application for BBM-D101 Injection, a gene therapy drug for Duchenne Muscular Dystrophy (DMD) developed by Belief BioMed, was approved by the U.S. Food and Drug Administration (FDA). In November 2024, this product received FDA Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD). Data indicates that BBM-D101 Injection is an adeno-associated virus (AAV) gene therapy drug independently developed by Belief BioMed. Administered through a single intravenous infusion using an engineered AAV vector, it delivers an optimized gene expression cassette to muscles throughout the body, aiming for a "one-time dosing, long-term efficacy" treatment for DMD.
 

The rapid development of cell and gene therapy in China will not only benefit patients domestically but also contribute Chinese wisdom and solutions to the global medical field. Industry experts note that the advancement of cell and gene therapy in China epitomizes the enhancement of the country’s scientific and technological innovation capabilities. With over 50% of global clinical trials, this is not just a numerical breakthrough but also a testament to the hard work and perseverance of Chinese researchers. In the future, as the innovation-driven development strategy continues to be implemented, China will achieve even greater milestones in the field of cell and gene therapy, making a larger contribution to global medical progress and human health.
 

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