Shanghai-Based Fengxun Bio's In Vivo CAR-T Therapy Set to Enter Clinical Trials, Potentially Slashing Cancer Treatment Costs from Millions to Tens of Thousands of Yuan

(Source: Shanghai Observer)

One injection can complete T-cell gene modification and activation in the patient's body, heralding a revolutionary shift in cell therapy from ex vivo to in vivo.

In recent years, the star anticancer drug CAR-T therapy has rewritten the history of hematological tumor treatment. However, its long ex-vivo preparation cycle and the high cost of millions per injection have deterred patients. To achieve product affordability through fundamental technological innovation, universal CAR-T and in-vivo CAR-T have successively entered the industry's field of vision.

Shanghai Fengxun Biotechnology, founded by one of the earliest teams globally to conduct in vivo CAR-T research, recently announced that its self-developed core in vivo CAR-T product has entered the critical preclinical stage. The candidate drug for hematological tumors, GI-001, will soon initiate IIT (Investigator-Initiated Trials). This more disruptive "universal" solution is expected to break the limitations of traditional CAR-T therapies, reducing drug costs to around ten thousand yuan, thereby benefiting more patients.

Turning the Human Body into a "Biological Factory," Restructuring Treatment Pathways

The technical route of traditional ex vivo CAR-T therapy involves extracting the patient's own immune T cells, equipping them with a navigation system called CAR (chimeric antigen receptor) through genetic engineering technology, which allows for precise recognition and attack of cancer cells. After substantial expansion in the lab, the modified cells are reinfused into the patient’s body to eliminate tumors, making the process extremely complex.

Instead of going through great efforts to modify cells outside the body, why not let the patient's own body complete the drug preparation? In vivo CAR-T technology bypasses complex ex vivo operations by directly delivering gene-editing tools into the patient’s body, turning the human body into a natural "biological factory" that transforms T cells into CAR-T cells in situ.

"In this way, the treatment process of CAR-T can be simplified into a single intravenous injection within 30 minutes, eliminating the need for patients to endure long waits and fundamentally avoiding the risk of disease progression that may occur during the traditional CAR-T therapy’s preparation period of 2 to 4 weeks," said Shi Kaikai, CEO of Fengxun Biotech.

According to the introduction, there are currently two major technological approaches for in vivo CAR-T: one is virus vector technology represented by lentiviruses, and the other is non-viral delivery technology represented by lipid nanoparticles. Fengxun Biotech has chosen the former. The company's scientific founder, Professor Cai Yujia from Shanghai Jiao Tong University, has been deeply involved in the field of virus vector modification and in vivo gene editing for many years. His laboratory is one of the earliest teams globally to initiate research on in vivo CAR-T technology based on lentivirus vectors.

To overcome the core bottleneck of in vivo CAR-T, Fengxun Biotech has independently developed three major core platforms. Through systematic innovation in precise targeting, efficient activation, and stable delivery, the in vivo CAR-T therapy becomes safer, more potent, and more controllable. For example, the company's self-developed viral vector can specifically target T cells, effectively avoiding off-target effects and non-target cell infection; the innovative design of a "co-stimulatory molecule" combination can efficiently activate resting T cells and promote their expansion, generating a long-lasting cytotoxic T-cell population; through serum resistance mutation modification, the stability and transduction efficiency of the viral vector in vivo are greatly improved, ensuring therapeutic efficacy while reducing drug toxicity.

Ripping Off the "Sky-High Price" Label, Companies from China and Abroad Rush to Enter the Market

"The million-yuan-per-shot" tag is difficult for traditional CAR-T therapies to shake off, and also a hurdle that patients find hard to overcome. Statistics show that among the 14 approved CAR-T products globally, the pricing of foreign products mostly ranges from $360,000 to $470,000, while the cheapest CAR-T product in China, "Yuan Rui Da," is priced at 999,000 yuan per shot.

The "astronomical cost" of CAR-T therapy mainly stems from its complex personalized manufacturing process—requiring a separate production line to be established for each patient, involving hundreds of processes and completed by multiple professional engineers. Industry insiders estimate that the production cost for a single CAR-T case is approximately 300,000 to 600,000 yuan, not including expenses for research and development, quality control, cold chain transportation, and other steps. Additionally, the lymphodepletion treatment before the patient's infusion is also quite costly.

In vivo CAR-T is regarded by the industry as the ideal universal CAR-T technology. Currently, more than a hundred companies in China have entered the field, and several multinational pharmaceutical enterprises have also set their sights on this area, rushing to join the competition. According to incomplete statistics, the total financing for in vivo CAR-T-related projects in China this year has exceeded 2 billion yuan. Fengxun Biotech, founded only half a year ago, has already completed seed and angel rounds of financing totaling nearly 60 million yuan, with well-known institutions such as CAS Star, Hanyuan Assets (Shanghai Jiaotong University's parent fund), and Legend Capital among its backers.

Shi Kaikai stated that as a truly "off-the-shelf product," the finished in vivo CAR-T drug can be directly stored in hospitals for immediate use; treatment also does not require lymphodepletion, and direct injection of the viral vector is sufficient, significantly reducing systemic treatment costs. He calculated for the reporter: if in vivo CAR-T successfully achieves commercialization, using a 200-liter viral production tank for mass production with 30 batches per year, each batch could meet the medication needs of hundreds of patients, and the treatment cost is expected to drop to around ten thousand yuan.

"Traditional CAR-T therapy has already brought hope to some cancer patients. Our goal is to make it more affordable and ensure timely access to effective treatment for more patients." Shi Kaikai revealed that the company's core pipeline product for treating hematologic tumors, GI-001, will soon initiate IIT clinical trials, with relevant trial data expected to be collected in the second or third quarter of next year. Meanwhile, GI-002, aimed at solid tumor treatment, is scheduled to enter the clinical exploration phase in the first half of next year. Additionally, the company is developing multiple cell therapy candidates for anti-fibrosis, anti-aging, and other applications, covering both oncology and non-oncology therapeutic areas.

Original Title: "'Million-yuan-per-shot' cancer drug may drop to ten thousand yuan! Shanghai's in-vivo CAR-T new therapy clinical trial is imminent"

Column Editor: Ren Quan

Source: Author: Wen Hui Bao, Liu Qi