Belief BioMed’s Co-Founder and CEO, Dr Jane Zheng, sets out an ambition to move gene therapy beyond scientific promise and into a scalable, globally accessible treatment model. Built on an AAV platform and supported by integrated manufacturing, the company is advancing from its haemophilia B proof of concept into broader indications, while navigating the realities of regulation, access, and cost. At the same time, it is exploring new approaches to partnerships and global delivery, reflecting a shift in how advanced therapies may reach patients worldwide.
How did Belief BioMed come into being, and what shaped your early strategic focus?
Belief BioMed was founded on a shared conviction that gene therapy could move beyond scientific promise and become a practical treatment option for patients, first in China and ultimately worldwide. My background in viral vector development and manufacturing complemented Professor Xiao Xiao’s more than three decades of work in gene therapy and adeno-associated virus (AAV) vector biology, and when we met, it became clear that we were aligned both in expertise and in ambition. We set out to build a company that could translate advances in the field into therapies that are not only innovative, but also accessible to patients who have historically had limited access to such treatments.
From the outset, we chose to focus exclusively on gene therapy and to anchor the company in an AAV-based platform, reflecting both the scientific maturity of AAV and our own experience in the field. AAV has become one of the most clinically validated delivery systems for in vivo gene therapy, and it offered a foundation on which we could combine vector engineering, tissue-specific delivery, and scalable manufacturing. Rather than treating AAV as a fixed technology, we saw it as a platform that could be refined through capsid engineering and optimisation to improve both efficacy and safety.
We therefore built Belief BioMed as an integrated gene therapy company, with capabilities spanning vector design, clinical development, and in-house manufacturing, allowing us to control the full process from early research through to potential commercialisation. This integrated model is central to our strategy, as it enables us to address one of the key barriers in the field, namely cost and accessibility, while maintaining consistency in quality and execution. Ultimately, our aim is to ensure that gene therapy can evolve into a sustainable and widely available treatment modality, rather than remaining limited to a small number of patients or markets.
In the recent years, AVV has faced some challenges as the platform of choice for gene manipulation biotechs. What role does AAV play within your platform, and how do you approach its optimisation?
AAV remains one of the most established delivery systems in gene therapy, largely due to its favourable safety profile, its predominantly non-integrating behaviour, and its ability to support sustained gene expression. At the same time, the field continues to address important limitations, including immune responses, off-target exposure, and dose-related toxicity. Our approach is to treat AAV not as a fixed solution, but as a platform that can be actively engineered and refined. At Belief BioMed, we have developed the CapsidX platform to design proprietary capsids with improved tissue specificity, enhanced transduction efficiency, and reduced immunogenicity, allowing us to achieve more precise and effective delivery than is typically possible with conventional AAV serotypes.
This focus on capsid engineering is closely integrated with optimisation at the level of the gene expression cassette. We work on promoters, intron insertions, and codon-optimised transgene sequences to ensure that gene expression is both strong and restricted to the intended tissue, thereby improving efficacy while limiting unwanted activity elsewhere. In our haemophilia programme, for example, the engineered AAV843 capsid is combined with a liver-specific promoter and an optimised factor IX (FIX-Padua) sequence to support targeted and sustained expression. By addressing both delivery and expression, we aim to improve the overall therapeutic window rather than relying on a single vector characteristic.
More broadly, this strategy reflects the combined experience of the founders in AAV biology, vector engineering, and manufacturing. We have built an integrated platform that spans vector design, clinical development, and in-house manufacturing, enabling us to maintain control over quality and scalability. This integration is essential to translating the biological advantages of AAV into clinically viable therapies that can be developed and produced consistently.
Why did you select haemophilia B as your first proof-of-concept indication?
Haemophilia B was a logical starting point because it is a monogenic disease with a clearly defined mechanism, which makes it particularly well suited to gene therapy. The condition is caused by mutations in the factor IX gene, so the therapeutic strategy is straightforward: delivering a functional copy of the gene can restore factor IX activity. This clarity allows for a more direct demonstration of proof of concept, especially in the early stages of development, where establishing a clear link between intervention and outcome is critical.
At the same time, the disease is associated with a significant and ongoing treatment burden. Patients typically require lifelong factor IX replacement therapy, administered through regular intravenous infusions, either on demand or as prophylaxis. Even with improved formulations, this remains demanding for patients and their families, particularly in settings where access to consistent prophylactic treatment has historically been limited. Gene therapy offers a different approach by enabling the patient’s own body to produce factor IX following a single administration, reducing reliance on repeated treatment.
Our lead programme, BBM-H901, was designed around this rationale. It combines an engineered AAV843 capsid with a liver-specific promoter and an optimised FIX-Padua transgene, with the aim of achieving sustained expression after a one-time treatment. Clinical data to date show reduced bleeding rates and durable factor IX activity over five-year follow-up, although continued observation remains important. Overall, haemophilia B provided a strong and well-defined context in which to establish clinical proof of concept, while addressing a meaningful unmet medical need.
How did you structure the clinical development of BBM-H901, and what does it reveal about current regulatory expectations for gene therapy?
The clinical development of BBM-H901 combined a structured programme with a pragmatic use of investigator-initiated trials (IITs) in China, which allowed us to generate early insights into safety, dosing, and initial efficacy while building a meaningful long-term follow-up dataset. These early participants now provide more than five years of follow-up in some cases, with sustained factor IX activity over time, offering an important perspective on durability. Meanwhile, it is essential to distinguish between exploratory data and the evidence required for regulatory approval.
Formal approval in mainland China, followed by approval in Macao, was based on a complete clinical programme that included phase I/II studies and a pivotal phase III study. In that phase III study, BBM-H901 met its primary endpoint, reducing the mean annualised bleeding rate to 0.60 within 52 weeks and achieving the mean factor IX activity within the therapeutic range. This one-year dataset formed the core of the registration package, supported by the broader clinical evidence and ongoing follow-up from earlier cohorts.
This reflects a broader principle in gene therapy development, where long-term follow-up is critical for understanding durability, but approval decisions do not necessarily require many years of data at the time of filing. Clinically, the results represent a strong functional response, enabling patients to move away from frequent replacement therapy towards a one-time treatment designed to support endogenous factor IX production. At the same time, continued observation remains an essential part of the field. To date, more than 5 years long-term follow-up data demonstrate durable efficacy.
Looking beyond China, we are assessing how this clinical package can support development in other markets, particularly in the United States. We are in communication with FDA, and our strategy is to continue engaging with regulators to determine the most appropriate pathway forward based on the full dataset.
How does your partnership with Takeda shape the commercialisation of BBM-H901, and how are you approaching access and international expansion?
Our collaboration with Takeda China reflects a deliberate and focused approach to the initial commercialisation of BBM-H901. As a first gene therapy of its kind in China, the product requires not only regulatory approval but also strong capabilities in rare disease management, physician engagement, and patient education, all of which are critical to adoption. Takeda brings established expertise in haemophilia and a well-developed local infrastructure, making it a natural partner for this stage. Under the agreement, Takeda holds exclusive commercialisation rights in mainland China, Hong Kong, and Macao, while we remain responsible for development and manufacturing, and continue to consider broader partnerships as we look beyond China.
The early launch phase reflects the realities of introducing a new therapeutic modality, where adoption depends as much on awareness and system readiness as on clinical data. Education remains a key factor for physicians, patients, and payers, and uptake will evolve progressively as familiarity with gene therapy increases. At the same time, interest is beginning to extend beyond China. The idea of international patients seeking treatment locally has been raised, and a mature pathway has not yet been established but is under exploration.
Access and affordability are central to how we think about long-term impact. While the treatment cost of our haemophilia B therapy is significantly lower than comparable gene therapies in the United States, pricing alone does not resolve the challenge, as reimbursement systems are still adapting to one-time, high-cost therapies. Together with Takeda, we are exploring alternative payment models, including supplementary insurance schemes, with initial progress at the city level, such as partial reimbursement in Beijing, Chongqing and Jiangxi Province, while broader inclusion continues to be discussed.
In parallel, we are evaluating international expansion in a measured way. In Saudi Arabia, BBM-H901 has received Orphan Drug Designation (ODD) from the Saudi Food and Drug Authority (SFDA), which provides a basis for further regulatory engagement but does not constitute approval. In the United States and Europe, we have obtained key regulatory designations, including orphan and Rare Pediatric Disease Designations (RPDD) from the US FDA and Advanced Therapy Medicinal Product classification from the EMA. These steps support the development pathway, and our focus now is to assess how our existing clinical and manufacturing package can be positioned to support future regulatory and commercial opportunities.
How are you expanding beyond rare diseases, and what underpins your strategy to develop gene therapies for chronic conditions?
Our pipeline reflects a deliberate progression from well-defined rare genetic diseases into selected chronic conditions where the unmet medical need remains high and the underlying biology is sufficiently understood to support a gene-based approach. Alongside programmes in haemophilia B, haemophilia A, and Duchenne muscular dystrophy, we are advancing early-stage assets in Parkinson’s disease, osteoarthritis, and cervical high-grade squamous intraepithelial lesion associated with high-risk human papillomavirus. Having completed initial clinical validation through investigator-initiated trials (IITs), all our common disease pipelines have accumulated certain clinical safety and efficacy data. Based on the positive data, we have advanced three common disease pipelines into the registrational clinical stage for further development.
The rationale for this expansion is both scientific and practical. From a biological perspective, we focus on indications where the mechanism of action is clear enough to guide a targeted intervention. In Parkinson’s disease, for example, our approach is to deliver genes involved in dopamine synthesis directly to the relevant brain region, with the aim of enabling sustained local production rather than relying on repeated pharmacological treatment. In osteoarthritis, we are exploring the potential for continuous intra-articular expression of anti-inflammatory proteins, reflecting a broader ambition to move towards disease modification, although this still requires advanced clinical validation.
At the same time, the strategy is shaped by the realities of access and sustainability. In China, although the rare disease population is meaningful, reimbursement pathways for high-cost innovative therapies remain less mature than in the United States or Europe. Chronic diseases, by contrast, involve larger patient populations and may allow for more scalable pricing models as manufacturing efficiencies improve and payment systems evolve. Underpinning this approach is confidence in the flexibility of our platform, where engineered AAV capsids and optimised gene constructs can be tailored to different tissues, allowing us to extend into new indications while recognising that each programme must be validated through its own clinical and regulatory pathway.
What are your immediate priorities as you scale the business and strengthen your global positioning?
Our immediate focus is to advance our pipeline while securing the partnerships needed to support the next stages of development, particularly as programmes approach phase III, where both capital and operational requirements increase significantly. In this context, collaboration with global pharmaceutical companies is a natural step to help bring the most promising assets forward. At the same time, we are working to strengthen our international visibility, with the aim of positioning Belief BioMed as a globally recognised gene therapy company and engaging more closely with investors in the United States and Europe, which remain central markets for advanced therapies.
This objective is closely linked to how we are evolving our organisational footprint. While our core operations are based in China, with main activities across Shanghai, Beijing, Suzhou, and Hong Kong, we are considering further expansion into key international hubs, including the United States, Europe, and Singapore. We do not yet have a formal office in the United States, but this is part of our longer-term plan. Our business development function is currently coordinated from China leveraging a highly focused team to ensure agile decision-making. Also we expect to expand it as our global engagement increases. Today, our team comprises around 230 people, reflecting both our development efforts and our investment in manufacturing.
Manufacturing remains a central pillar of our strategy and an important differentiator as we scale. We have established a commercial-scale gene therapy facility in Shanghai, covering approximately 15,000 square metres, with multiple production lines and bioreactor capacity of up to 2,000 litres. The site has been built in line with GMP standards across China, the United States, and Europe, and has already undergone regulatory inspection in China to support both clinical and commercial supply. It has also been reviewed by partners such as Takeda and AskBio, whose teams have assessed our CMC systems and overall readiness. As we move towards broader international development, further regulatory inspections, including by the US Food and Drug Administration, would form a standard part of the process, and we are preparing accordingly.
What message would you like to share with global pharmaceutical partners and investors?
Our focus is to ensure that gene therapy evolves from a scientific breakthrough into a treatment option that is both effective and accessible at scale. We believe this is a shared objective across the industry, and it creates a strong basis for collaboration. At Belief BioMed, we bring core strengths in vector engineering, new drug (gene therapy) research and development, platform development, and manufacturing, while global pharmaceutical partners contribute experience in clinical development, regulatory engagement, and patient access. Bringing these capabilities together is essential to move gene therapy forward in a way that is both scientifically robust and commercially viable.
At the same time, the nature of gene therapy calls for a different perspective on access. As a one-time treatment with the potential for long-term benefit, it does not fit easily into traditional delivery models, and this opens the door to more centralised approaches, including the possibility of patients travelling to specialised centres for treatment. While this remains an evolving concept, it reflects a broader shift in how advanced therapies may be delivered in the future. Our aim is to combine innovation, cost-efficient manufacturing, and global collaboration to support this transition and extend the reach of gene therapy to a wider patient population.
