On August 20, Takeda China and Biocad jointly announced that both parties had officially terminated their commercial collaboration on the hemophilia B gene therapy drug, pomalidomide (trade name: Biocad).

The collaboration between the two parties began in October 2023. Pemigatinib injection is the first AAV gene therapy drug approved for marketing in China, with hemophilia B (B-type hemophilia) as its indication—the second most common type of hemophilia. Compared to traditional therapies, the most notable feature of this treatment is its single intravenous injection administration.

This transaction also marks China's first gene therapy drug successfully developed and produced by a domestic enterprise, with commercialization rights licensed to an external party. Whether from its clinical value or its exemplary significance for China's CGT commercialization, popadokig was once highly anticipated.

However, technological value ultimately must pass the test of commercialization.

Since its approval for market launch in April 2025, the commercial performance of Patisiran has been less than optimistic. On one hand, the Chinese market's payment capacity for high-priced innovative drugs, particularly one-time gene therapy products, remains limited. On the other hand, Takeda itself has been continuously adjusting its business structure. In October 2025, Takeda Pharmaceutical Company announced a complete halt to gene-cell therapy-related operations, followed by a workforce reduction plan.

Amidst the convergence of multiple factors, this once-celebrated industry collaboration ultimately reached its end.

Regarding the termination of this collaboration, Dr. Xiao Xiao, Co-founder, Chairman, and Chief Scientist of Belief Biopharma, provided an exclusive response to Tongxixi. Xiao emphasized that the decision to end the partnership was a proactive business decision made by both parties based on strategic focus.

However, the termination of the collaboration does not imply a suspension of the commercialization of Popadakji.

Xiao Xiao told Writenews that during the collaboration, Belief Pharmaceuticals and Takeda China jointly worked to promote clinical awareness, patient accessibility, and affordability of hemophilia B gene therapy in China. The clinical experience and patient management insights accumulated during this period will continue to provide valuable references for the development of hemophilia treatment in China. Next, Belief Pharmaceuticals plans to independently advance the commercialization of Bococizumab.

Against the backdrop of an imperfect existing payment system, the commercialization of high-cost rare disease drugs must balance sustainability and accessibility while maximizing operational efficiency. In the future, belief will actively explore various approaches to address challenges in the commercialization process, such as charitable drug donations

For the Chinese CGT industry, how to commercialize high-priced innovative therapies remains a challenge without a standard solution.

Even so, practitioners including Xiao Xiao and Belief Biopharma continue to choose to stay and explore further. They believe the commercialization challenges of CGT are not insurmountable but require finding a path more suited to the Chinese market.

Xiao Xiao
Co-founder, Chairman, and Chief Scientist of Belief Pharmaceuticals
 

01. China's first AAV gene therapy stands at a crossroads

When the contract was signed in 2023, the Chinese cell and gene therapy (CGT) sector was at its peak.

According to the Insight database, the trading enthusiasm in the global CGT field continues to rise. From 2020 to 2023, there have been at least 35 CGT transactions with transaction amounts exceeding $500 million. Capital support is also driving the rapid development of CGT technology and industry.

Takeda had maintained a strong interest in gene therapy at that time. Prior to that, Takeda had been investing in the AAV gene therapy field for many years. After seeing the clinical data published by Bopartacogene, Takeda ultimately decided to acquire the commercialization rights of the product in Greater China.

In the industry environment at that time, this transaction was regarded by the industry as a landmark case of commercial cooperation between China's CGT and multinational pharmaceutical companies.

The clinical data of bupropion is indeed impressive.

According to the official website of Belief Pharmaceuticals, the results of the BBM001-CLN1001 study showed that after 26 hemophilia B patients received 5 × 1012 vg/kg of bopatecogene therapy for 52 weeks, the annualized bleeding rate (ABR) decreased to 0.6, significantly lower than the superior efficacy threshold of 5.0.

In terms of safety, the results of Phase I/II and Phase III clinical studies on the treatment of adult hemophilia B with bopatide, as well as the long-term follow-up results of investigator-initiated clinical trials (IIT), have been published in the internationally authoritative journal Nature Medicine.

The long-term follow-up results of more than 5 years from IIT showed that 90% of the subjects maintained a continuously high and stable expression of FIX, without experiencing any bleeding events. The annualized bleeding rate (ABR) was 0, and the replacement therapy with exogenous FIX products was completely discontinued. During the study period, there were no deaths, serious adverse events, thromboembolic events, or adverse events with positive FIX inhibitors. Additionally, there were no adverse events related to abnormal transaminases during the long-term follow-up.

Hemophilia B is an X-linked recessive hereditary bleeding disorder caused by mutations in the coagulation factor IX (FIX) gene, leading to a significant decrease in the plasma FIX content or activity.

Bopartacogene utilizes recombinant adeno-associated virus (rAAV) vectors to deliver optimized human FIX gene to patients' liver cells through intravenous administration. The gene expression system of the host cells is utilized to continuously produce FIX and secrete it into the bloodstream, thereby exerting a procoagulant effect.
For a long time, the standard treatment for hemophilia B has primarily relied on coagulation factor replacement therapy. Patients often require lifelong medication, and those with severe cases also need long-term prophylactic infusions. Frequent venipuncture has also become a lifelong treatment burden for patients.

Gene therapy offers another possibility: through a single intravenous infusion, patients can acquire the ability to continuously express FIX, thereby achieving "one-time administration, long-term benefit," potentially eliminating the need for lifelong frequent infusions.

From the perspective of clinical value, this is undoubtedly an attractive technology. However, when the technology moves towards commercialization, reality soon sets in.
The initial pricing of Bopida Keqi after its launch was 93,000 yuan per single bottle of 2ml, and the specific dosage needs to be calculated based on the patient's weight. After charitable assistance, the maximum payment for patients still reached 2.79 million yuan.

There exists a huge gap between high pricing and patients' affordability.

According to the report titled "Payment Plan and Global Strategy for Innovative Therapies for Hemophilia" released by the Beijing Pain Challenge Public Welfare Foundation, nearly 90% of patients are willing to choose gene therapy. However, the median willingness to pay for gene therapy among patients is only 50,000 to 80,000 yuan, which is significantly lower than the pricing level of Bopapadak.

In June 2026, the price of Bopida Keqi was reduced by over 50%, bringing the cost for patients down to 1.39 million yuan or less. Following the price reduction, market feedback also began to change.

"After the price reduction, many patients have inquired about medication-related matters from the company," said Xiao Xiao. He still holds hope for the future commercialization of Bopartap. "We may soon see news about the first prescription being issued."

02. The commercialization of millions of CGTs

After taking back the commercialization rights of Vapidac from Takeda, Faith Pharmaceuticals decided to independently promote its sales business.

Compared to handing over the product to a partner, Belief Pharmaceuticals takes charge of commercialization itself, enjoying greater autonomy in pricing, charitable assistance, and patient support.

For a one-time therapy with limited patient numbers and high treatment prices, this flexibility may be particularly important.

"The sales logic for rare diseases is different from that of ordinary drugs, and there is no need to build a sales team of hundreds or thousands of people."

Instead of establishing a large sales team, it is better to focus on a limited patient population and core diagnosis and treatment centers, and invest limited resources into more precise patient outreach and medical promotion.

The payment side is another hurdle that Faith Pharmaceuticals needs to focus on breaking through next. From a broader industry perspective, the payment side is becoming an increasingly crucial link in the innovative drug industry chain.

In the 2026 government work report, biomedicine was listed as an emerging pillar industry for the first time, and the government will continue to increase support for the biomedicine industry and the research and development of innovative drugs. However, the transition of new drugs from research and development to the market does not mean that the industrial chain has truly completed a closed loop. If the payment system cannot accommodate it, resulting in difficulties in reaching patients with new drugs, the value of innovation will be difficult to transform into industrial value, which may also affect the willingness of enterprises to continue investing in research and development.

Especially for high-priced, one-time CGT therapies, whether the payment side can be connected is not just a matter of commercializing a single product, but also relates to whether the innovative achievements of the entire industry can be truly implemented.

Currently, Belief Pharmaceuticals is actively promoting the inclusion of Bopartide in the reimbursement scope of various local health insurance programs. Xiao Xiao introduced that several provinces and cities have already included Bopartide in the reimbursement scope of their health insurance programs, including Shanghai's "Shanghai Health Insurance".  

Meanwhile, Belief Pharmaceutical also plans to make another attempt to enter the commercial health insurance innovative drug catalogue. In 2025, Bopida Kiji made an attempt to enter the first edition of the commercial health insurance innovative drug catalogue, but ultimately failed to be selected.

In that year, a total of 121 drugs passed the formal review, but only 19 drugs from 18 companies were selected, with a pass rate of less than 16%. Among the selected drugs, all five CAR-T therapies made the list, as well as drugs for rare diseases such as neuroblastoma and Gaucher's disease, and drugs for Alzheimer's disease.

Xiao Xiao said that Bopidaki has submitted an application this year, "Although we don't know the outcome, it's something we have to strive for."

The "Several Measures on Supporting the High-quality Development of Innovative Drugs" clearly states that drugs included in the commercial insurance innovative drug catalogue will not be included in the basic medical insurance out-of-pocket rate indicators and the scope of monitoring for alternative varieties selected in centralized procurement, providing support for commercial insurance to expand its coverage and optimize services.

In addition to expanding reimbursement coverage, the payment model itself also requires innovation.

Xiao Xiao is actively discussing payment schemes for gene therapy with experts in pharmacoeconomics, hospital representatives, patient organizations, insurance companies, and other stakeholders.

"For example, paying by installments based on treatment efficacy is a direction worth exploring for Bopartacogene. This can alleviate the burden of one-time payment for patients, and hemophilia has objective indicators such as coagulation factor concentration to observe treatment efficacy."

In his view, the key to the efficacy payment model lies in finding quantifiable and long-term trackable efficacy indicators. Indicators such as FIX levels and bleeding rates in hemophilia are relatively clear and provide a basis for exploration. However, not all CGT products can replicate this path, as some, such as neurodegenerative diseases, lack quantifiable efficacy evaluation indicators.

There is another underlying issue, which is the mismatch between the current medical insurance payment logic and one-time treatment.

Xiao Xiao pointed out that currently, medical insurance payments are typically based on annual treatment costs, which is not favorable for many one-time treatments such as CGT therapies.

Taking Bopida Keqi as an example, despite the price reduction, the one-time treatment cost of 1.39 million yuan still seems like an astronomical figure. If only comparing the annual cost, it is obviously difficult to reflect the long-term economic value of one-time gene therapy; but if the treatment cycle of the patient for the next few decades is taken into account, the conclusion may be completely different.

According to the 2024 estimates in the "Leopard Head" series report, based on the AHA clinical diagnosis and treatment guidelines, first-line medications for hemostasis treatment include drugs such as human coagulation factors and prothrombin complex concentrate. Calculated based on an average patient weight of 60kg, the annual medication cost for hemophilia patients can reach 630,000 yuan.

How to clearly calculate and explain the trade-off between "one-time high costs" and "long-term reduction in treatment costs" may be the key for CGT to further open the doors to medical insurance and commercial insurance.

Bobatacchi is now standing at a crossroads.

It was once a technological milestone in the treatment of hemophilia, and now it has become a representative example of the commercialization dilemma faced by high-priced innovative therapies.

From cooperation between multinational pharmaceutical companies to independent advancement by local enterprises, what has changed is not only the commercialization entities, but also the process of an industry seeking answers: for high-value CGT therapies, what kind of payment system does China need to truly transform technological value into patient accessibility?

Bobataki has not yet provided a final answer. But at least, Faith Pharmaceuticals is still trying.