Cancer remains the leading cause of death in Taiwan. Over 90% of global cancer patients suffer from solid tumors, which are considered the most challenging frontier for CAR-T cell therapy. China Medical University Hospital (CMUH) announced yesterday (March 10) its successful collaboration with Ever Supreme Bio Tech to develop the world’s first "EXO 001 Targeted Exosome Platform." This platform can reprogram T-cells directly within the body to generate multi-target nano-antibody CAR-T cells, demonstrating significant efficacy—and even complete tumor clearance—in multiple solid tumor animal models.

Dr. Der-Yang Cho, President of CMUH, pointed out that current CAR-T therapies (whether autologous or allogeneic) rely on tedious and time-consuming ex vivo manufacturing. This delay is often fatal for patients with rapidly progressing solid tumors. Furthermore, existing treatments face limitations such as immune rejection, manufacturing failures, and prohibitive costs. The core design of EXO 001 allows exosomes to act as "in vivo immune training vehicles," representing an advanced evolution of the CAR 001 currently in clinical trials.

During treatment, the exosomes are administered intravenously and circulate to key immune organs, such as the spleen, where they deliver CAR.BiTE genes to T-cells. This completes the immune reprogramming within the body, training T-cells to transform into multi-target CAR-T cells. These cells then infiltrate solid tumors to kill cancer cells and dismantle the tumor microenvironment—essentially "completing training in vivo for immediate combat." This approach combines the immune compatibility of autologous CAR-T with the "off-the-shelf" convenience of allogeneic CAR-T.

Dr. Cho explained that the research team confirmed in mouse models for colorectal, pancreatic, brain, and ovarian cancers that intravenous injection of EXO 001 generated CAR-T cells in vivo. These cells successfully penetrated the tumor microenvironment, significantly inhibited tumor growth, and extended survival by 2 to 3 times. Some animals showed complete disappearance of cancer cells with no long-term recurrence. Additionally, using exosomes as delivery vectors offers higher biocompatibility and safety compared to viral or synthetic materials, reducing the risk of anti-drug antibodies and immune-related complications.

Huang Wen-liang, General Manager of Ever Supreme Bio Tech, highlighted several advantages for clinical and industrial scaling:

1. Stable Quality from a Single Cell Source: Minimizes donor variability, facilitating standardization and quality control.
2. Mass Production & International Compliance: Produced in a fully closed, automated process compliant with U.S. FDA regulations for cellular products.
3. Off-the-Shelf Availability: Eliminates the wait time associated with ex vivo cell culture, crucial for patients with aggressive solid tumors.
4. Cost-Efficiency: Significantly lowers production costs compared to traditional autologous CAR-T, enhancing clinical accessibility.
5. Platform Potential: Capable of carrying different CAR genes, nucleic acids, or small molecule drugs for multiple indications.
6. Long-term Immune Memory: Animal studies show EXO 001 can induce CAR-T cells with immune memory characteristics, potentially leading to more durable anti-cancer effects.

This research was accepted for publication in the prestigious international journal Advanced Science in January 2026 and has secured patents in the U.S. and other countries. The technology transfer is complete, and process development and clinical trial preparations are underway. Clinical trials are expected to launch as early as next year for patients with late-stage colorectal, pancreatic, brain, and ovarian cancers, bringing new hope to terminal cancer treatment.

Resource: 中醫大附醫攜長聖 推EXO 001活體內CAR-T