According to industry analysis of the world's top 10 most expensive gene therapies, a total of 77 competing products are currently in development.

Of these, 48 originate from China—more than twice the number from the U.S. and seven times that from Europe. This means behind every gene therapy priced at millions of dollars, there are at least seven domestic challengers waiting in the wings.

Source: Endpoints

Each name on this list of the most expensive drugs represents the pinnacle of the pharmaceutical industry: Zynteglo ($2.8 million per dose), Elevidys ($3.2 million), Roctavian ($2.9 million), and Casgevy ($2.2 million). The most expensive therapy on the list, Lenmeldy ($4.25 million), has three Chinese competitors in development, with no pipeline projects from the U.S. or Europe targeting this indication.

The only therapy where U.S. and European pipeline projects outnumber China's is Lyfgenia ($3.1 million) for sickle cell disease—yet even here, Chinese companies account for over one-third of all pipeline projects in this field.

China's regulatory pathway provides unique advantages for the rapid development of gene therapies. According to McKinsey research, patient recruitment time for clinical trials in China is only half the global average, with per-patient costs reduced by approximately 50%.

More importantly, domestic pricing mechanisms force cost control. In April 2025, Belief Therapeutics received approval for China's first fully localized gene therapy, Xinjiuning (for hemophilia B), priced at approximately $350,000—just one-tenth of comparable U.S. competitors. This price advantage marks a critical step in transforming gene therapy from a "luxury for wealthy nations" to an "accessible treatment option."

This competitive landscape has emerged precisely as Western gene therapy faces a fragile moment.

BioMarin announced in February it would withdraw its hemophilia A therapy Roctavian after failing to find a buyer. UniQure's hemophilia B therapy Hemgenix has seen slow market adoption, treating only 75 patients in three years post-approval. Most other first-generation gene therapies remain limited to wealthy nations or have failed to meet clinical expectations.

Global payers appear to be continuously resisting multi-million-dollar single-course pricing, and the economic logic that once supported gene therapy's high prices—replacing lifelong chronic disease management with a single treatment—has yet to be fully realized.

Against this backdrop, China's "low-cost, high-efficiency" model is redefining the commercial viability and patient accessibility of gene therapy.

If the rise of gene therapy represents the spearhead breakthrough of China's biomedicine industry, then the broader pharmaceutical sector is witnessing a systemic transformation.

Years ago, China’s biggest contribution to the global pharmaceutical industry was active pharmaceutical ingredients (APIs) — the behind-the-scenes work of supplying basic chemicals for Western innovative drugs. Back then, the West regarded China merely as a manufacturing powerhouse, skilled in generics and producing low-cost copies. The idea of “Chinese innovation” in pharmaceuticals was barely taken seriously.

Today, the landscape has been completely upended.

According to Goldman Sachs data, roughly one-quarter of innovative drug candidates in active R&D worldwide originate from China. More significantly, 46% of new drug molecules entering clinical trials in the first half of 2025 came from Chinese pharmaceutical companies, compared with only about 17% a decade ago. In licensing deals, China accounts for approximately half of the global transaction value and 26% by deal volume.

A decade ago, big pharma seeking the next breakthrough molecule would turn to U.S. or European biotechs. Today, major pharmaceutical companies are just as likely to license molecules from Chinese firms.

In oncology — a critical therapeutic area — Chinese pharma’s advantages are particularly pronounced. As of November 2025, 54 oncology-focused licensing deals had been completed that year. Over the past two and a half years, Chinese pharmaceutical companies have accounted for roughly 70% of global ADC R&D programs and about 60% of bispecific antibody R&D projects.

Source: Goldman Sachs (as of November 2025)

Goldman Sachs analysts note that global pharmaceutical companies increasingly view China as a source of innovation. They observe that China possesses unique advantages in molecular engineering capabilities. “Given China’s cost-efficiency advantages in early-stage R&D and its extensive exploration of drug component combinations, we believe engineering expertise will continue to accumulate among Chinese pharmaceutical firms.”

Chinese pharmaceutical companies typically conduct Phase I clinical trials domestically at a lower cost, then license the programs to Western drugmakers, who bear the expense of U.S. trials and bring the drugs to market—an arrangement that benefits both parties.

At the recently concluded 2026 China Development Forum, global pharmaceutical giants including Eli Lilly, GSK, Roche, AstraZeneca, and Novo Nordisk gathered in Beijing. Emil Kongshøj Larsen, Executive Vice President of Novo Nordisk, stated plainly that China has long evolved from a destination for innovation to a launchpad for it, with its “China Co-Innovation” program achieving simultaneous innovation with the rest of the world.

With an investment of over 100 billion yuan announced this January, AstraZeneca is accelerating its full-chain localized layout: establishing a commercial production and supply base for cell therapies in Shanghai, a radiopharmaceutical conjugate manufacturing facility in Guangzhou, co-building an AI drug R&D center with Tsinghua University, and forming a joint venture with CSPC. These moves have transcended the traditional “license-in” model and evolved into deep co-creation under the paradigm of “In China, For the World.”

Behind the collective actions of MNCs lies recognition of China’s innovation ecosystem. Severin Schwan, Chairman of the Board of Directors of Roche Group, highly commended that the Chinese government’s sustained investment in basic science and higher education has built a world-class research environment; progress in the regulatory system in terms of approval efficiency and intellectual property protection provides institutional safeguards for high-quality industrial development.

Meanwhile, these companies are also participating in the co-construction of China’s 15th Five-Year Health Blueprint through policy recommendations. Eli Lilly submitted a policy proposal to the forum, calling for the integration of weight management into national planning and promoting the inclusion of innovative drugs in medical insurance and the Essential Drugs List; GSK proposed taking the functional cure of hepatitis B as a breakthrough to drive the transition from “passive treatment” to “proactive health” and improve the full-life-cycle prevention system.

Why now? What changes have made China such a prominent source of new drugs?

A widely held view points to the 2015 regulatory reforms as the catalyst for the current growth in China’s innovative drug discovery, with an immediate impact. In 2017, the number of first-in-human clinical trial applications for innovative drugs surged nearly 80% year-on-year. After China joined the ICH, its drug development system became aligned with Western regulatory authorities.

But the more significant change may lie in the drastically shortened time to initiate clinical trials, especially first-in-human (Phase I) trials.

Before testing a drug in humans, pharmaceutical companies must submit an Investigational New Drug (IND) application to their national regulatory authority. In 2018, the NMPA adopted a “deemed approval” system for clinical trials—if the regulator raises no objections within 60 days of IND submission, the company may proceed with the trial. As a result, the average approval time for IND applications dropped from 501 days before the reform to just 87 days afterward.

The 2015 reforms coincided with a wave of returnee biotech talent who had been trained at top Western universities and companies, bringing not only scientific expertise but also a profound understanding of modern drug development. Decades of manufacturing experience had strengthened the industrial foundation when these talents returned to China.

Furthermore, CROs such as WuXi AppTec have built strong capabilities while serving multinational clients, providing infrastructure for today’s emerging biotech companies. Some CROs even use revenue from their services to fund internal drug discovery programs. Domestic discovery labs have matured to match U.S. facilities in many fields but operate at lower costs—scientists’ salaries are typically just one-quarter of those of their Western counterparts.

According to a McKinsey report, the post-reform boom in venture capital in China further fueled growth, triggering a wave of domestic company formation. Venture capital investment in China’s biotech sector rose from $4 billion in 2015–2017 to $12 billion in 2018–2020. Factors such as the return of talent, a solid industrial base, and funding have enabled China’s biopharmaceutical industry to move up the value chain and assume a globally competitive position in drug discovery.

If any single factor is most critical, it is likely the speed of Chinese pharmaceutical companies.

Take Legend Biotech as an example. Founded in 2014, its programs entered clinical trials in 2016, and it struck a deal with Johnson & Johnson in 2017. In contrast, Western biotech companies typically take 3–6 years to meet IND requirements and advance new drugs into clinical stages.

Western drug R&D programs progress slowly partly because companies practice extensive risk aversion to conserve capital; they may hesitate to run multiple experiments in parallel due to cost concerns, whereas Chinese pharma can afford to pursue multiple approaches simultaneously.

Some argue that the rise of Chinese pharma may not be sustainable, citing a decline in venture capital. As investors grow cautious, Chinese drugmakers may be more eager than usual to sell assets at a discount, with Western firms as ready sources of capital. However, considering other favorable factors, this appears to be a temporary fluctuation.

For now, China’s emergence as the global leader in the volume of new drug R&D seems almost inevitable. Licensing deals represent the primary revenue stream for biotech companies to fund operations and research—and that revenue is now flowing to China.