Fangtuo Bio, a company specializing in adeno-associated virus (AAV) vector gene therapy, officially submitted its A1 listing application to the main board of the Hong Kong Stock Exchange, becoming the first AAV gene therapy company to apply for listing in the Hong Kong stock market. UBS and Cathay Securities are acting as joint sponsors.

01
Differentiated pipeline layout

Founded in 2019, Fangtuo Bio is a clinical-stage gene therapy company with an international footprint. It has built a product portfolio of eight self-developed drug candidates, primarily focusing on ophthalmic and cardiovascular disease areas with significant unmet clinical needs. Among them, two core products (FT-002 and FT-003) and one key product (FT-001) have entered the clinical stage , while the remaining five are in the preclinical and early-stage development stages.

FT-002 , used to treat X-linked retinitis pigmentosa (XLRP), is the only therapy of its kind in China to have entered the clinical stage. XLRP is one of the most common and severe inherited retinal diseases, and currently there are no approved targeted therapies globally, representing a significant unmet clinical need. FT-002 received Orphan Drug Designation and Fast Track Designation from the US FDA in 2024. Currently, FT-002 is undergoing Phase II clinical trials in China and has received FDA approval to advance to Phase II clinical trials in the United States.

FT-003 is used to treat neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME), and is the only intravitreal gene therapy in the world to enter a Phase II clinical trial for DME.

In terms of patient population size, the market potential of this sector is extremely considerable. Data from 2024 shows that there are approximately 4.1 million nAMD patients and 7.6 million DME patients in China, and 1.6 million nAMD patients and 2 million DME patients in the United States. This large patient base constitutes a solid foundation for market demand.

Current standard treatment primarily relies on frequent intravitreal injections of anti-VEGF drugs, typically every 4 to 8 weeks. This approach places a significant psychological burden on patients and has poor long-term adherence. From an economic perspective, the cumulative cost of anti-VEGF therapy can reach as high as $304,000 in the United States and approximately 403,000 RMB in China, making it prohibitively expensive. More importantly, frequent intravitreal injections increase the risk of complications such as ocular fibrosis, retinal scarring, and geographic atrophy, further impairing long-term visual outcomes.

FT-003 has the potential to simplify the treatment burden to a single outpatient injection. Through an engineered AAV2 variant capsid, FT-003 can efficiently and broadly transduce various retinal cells, achieving robust and sustained expression of aflibercept in the eye. This, in turn, fundamentally inhibits pathological angiogenesis, reduces vascular permeability, and prevents ocular vascular leakage. Currently, FT-003 has initiated Phase II trials in both China and the US for nAMD and DME, with early data showing promising efficacy and safety signals.

FT-001 is used to treat inherited retinal diseases (IRD) caused by mutations in the RPE65 gene. In 2017, the US FDA approved Luxturna as a treatment for this disease, marking the first clinical validation of the efficacy of RPE65 gene therapy. Luxturna is currently priced at approximately $850,000 and has not yet been approved for marketing in China, indicating a significant unmet need. FT-001 received Breakthrough Therapy Designation from the China Center for Drug Evaluation (CDE) in June 2025. Phase I/II clinical trials have been completed in China, and the Phase III clinical trial protocol was approved by the CDE in September 2025.

In terms of targeted therapy for gene therapy drugs, ophthalmic diseases are the most prominent target area for gene therapy drugs in clinical development in both the United States and China. According to data from PharmNet, there are currently over 30 gene therapy drugs for ophthalmic diseases in clinical development in both China and the US.

Beyond ophthalmology, Fangtuo Biotechnology's research and development has extended to the cardiovascular field, a treatment area with a massive patient base. Cardiovascular disease is the leading cause of death worldwide, and traditional drugs primarily focus on alleviating symptoms rather than addressing the root cause of the disease. Gene therapy, on the other hand, offers a treatment pathway that corrects or alters the pathophysiological mechanisms of the disease at its source.

Fangtuo Biotech's pipeline includes two cardiovascular gene therapy candidates for the treatment of hypertrophic cardiomyopathy (HCM) caused by MYBPC3 gene mutations and arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by PKP2 gene mutations. Among them, FT-017 is the first AAV gene therapy in China to enter clinical trials for MYBPC3 mutation-related HCM.

The company's R&D and manufacturing capabilities rely on two proprietary platforms. The EXACTE™ R&D platform integrates advanced technologies and methods across the entire chain, from vector design and engineering to candidate drug screening and preclinical validation. The AAVANCE™ manufacturing platform , based on the Sf9 insect cell expression system, has an empty shell rate of less than 1%.

02
Financial performance

As a clinical-stage company that has not yet launched any products, Fangtuo Biotech's financial situation exhibits typical characteristics of a biotech company: continuous losses and tight cash reserves .

The prospectus shows that the company's pre-tax loss narrowed from approximately US$35.86 million in 2023 to approximately US$26.46 million in 2024, and further decreased to approximately US$13.31 million in the first nine months of 2025. The continuous narrowing of the loss reflects, to some extent, the effectiveness of the company's cost control and resource optimization.

In terms of R&D investment , the company has shown a contraction trend in recent years: R&D expenditure was US$27.6 million in 2023, dropped to US$20.6 million in 2024, and further contracted to US$11 million in the first nine months of 2025.

On the revenue side , since no products have yet been commercialized, Fangtuo Biotechnology's revenue relies entirely on government subsidies and other income. Government subsidies and other income amounted to US$904,000 in 2023, decreasing to US$617,000 in 2024, and further decreasing to US$451,000 in the first nine months of 2025.

As of September 30, 2025, the company's cash and cash equivalents amounted to only US$9.684 million, a significant decrease from US$13.5 million in 2023. In the first nine months of 2025, the company experienced a net cash outflow of US$10.4 million from operating activities. According to the prospectus, based on the current cash burn rate, the existing funds can only sustain the company's operations for approximately 17 months. This indicates that the core driving force behind this Hong Kong listing is to provide "ammunition" for subsequent key clinical research, alleviate significant financial pressure, and ensure uninterrupted R&D progress.
03
What are the prospects?

The global gene therapy market is experiencing rapid growth. The market size reached $2.8 billion in 2024, with a compound annual growth rate of 312.9% from 2020 to 2024. It is projected to exceed $32 billion by 2035.

China's gene therapy market is also poised for rapid growth, about to enter a golden age of explosive expansion. It is projected to grow at a CAGR of 53.4% from 2025 to 2035, reaching a market size of RMB 11.6 billion by 2035. In December 2025, the National Healthcare Security Administration released the "Commercial Health Insurance Innovative Drug Catalog," covering 19 innovative drugs with significant clinical benefits but not covered by public medical insurance, including several gene therapy drugs. This is expected to further drive the growth of the gene therapy market.

Among numerous gene therapy technologies, AAV gene therapy stands out as one of the most promising directions due to its unique advantages, particularly demonstrating enormous potential in the treatment of rare genetic diseases. Currently, several AAV gene therapy products have been approved for marketing globally, covering areas such as spinal muscular atrophy (SMA) and inherited retinal diseases.

In recent years, large pharmaceutical companies have made significant inroads into the AAV gene therapy field through mergers and acquisitions or licensing collaborations. For example, in October 2025, Eli Lilly acquired Adverum Biotechnologies for $262 million, gaining access to the latter's AAV gene therapy candidate Ixo-vec (targeting nAMD), which was in Phase III clinical trials. In November of the same year, Eli Lilly acquired the global exclusive rights to AAV-AIPL1 from MeiraGTx Holdings through a $75 million upfront payment plus $475 million in milestone payments. In October 2025, Otsuka Pharmaceutical acquired the exclusive rights in the Asia-Pacific region for 4D-150 (targeting nAMD and DME) from 4D Molecular Therapeutics for $471 million.

Despite the industry buzz surrounding AAV gene therapy due to its groundbreaking therapeutic potential, its development has not been without its challenges.

As clinical research deepens and commercialization progresses, inherent safety issues of AAV vectors, such as hepatotoxicity and immune rejection, continue to surface. Coupled with commercialization challenges like exorbitant pricing, concerns about the industry's future are gradually emerging. Pharmaceutical giants like Johnson & Johnson and Takeda have chosen to divest their AAV gene therapy assets, prompting reflection within the industry.

Pfizer 's hemophilia B gene therapy, Beqvez, is a typical example. This gene therapy drug was approved by the US FDA in 2024 for the treatment of hemophilia B, with a price tag of up to $3.5 million per dose. However, after nearly a year on the market, it failed to attract any patients to undergo commercial treatment and was eventually forced to withdraw from the market.

Johnson & Johnson's gene therapy bota-vec for X-linked retinitis pigmentosa also failed to meet its primary endpoint in a Phase III clinical trial, leading to the termination of this gene therapy, which was acquired for a total transaction price of $415 million.

In September of this year, Biogen announced that it had terminated its gene therapy program using AAV vectors and would instead focus its resources on treatment modalities that are most likely to achieve better therapeutic results.

Most approved therapies target extremely rare diseases affecting a small segment of the patient population, thus requiring high pricing to recoup the substantial upfront investment. However, healthcare systems and payers face significant difficulties in bearing these high costs, resulting in fragmented and limited reimbursement policies that severely restrict patients' actual accessibility to these innovative therapies.

04
Conclusion

As a revolutionary technology poised to fundamentally alter the paradigm of disease treatment, gene therapy's long-term development potential has become an industry consensus. Currently, adeno-associated virus (AAV) remains the mainstream vector for gene therapy applications, and research into its delivery efficiency, targeting, and safety is continuously advancing. Fangtuo Biotech's differentiated product pipeline and its impressive shareholder lineup fully demonstrate the capital market's recognition of the long-term value of the AAV gene therapy sector.

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