Keeping US biotech ahead of China could help patients with rare diseases: experts


The growth of China's biotech sector has been astonishing. Beijing is pumping money into the industry, supporting research efforts and helping launch a new wave of labs and incubators in the country. This is a problem for the American biotechnology industry and also affects rare disease patients waiting for a cure.

Among the experts speaking out against China's growing influence in the biotech sector is John Crowley, executive director of the lobby group Biotechnology Innovation Organization (BIO).

Crowley is something of a rock star in the rare disease community. His story is as incredible as it is inspiring.

When Crowley and his wife were told that their two young children had Pompe disease, a deadly genetic disorder, Crowley left his job in marketing to try to find a cure. He teamed up with a researcher who was working at Pompe and founded a company that eventually developed a treatment to save the lives of his children and thousands of others.

If it sounds like the plot of a movie, it is. Pulitzer Prize-winning journalist Geeta Anand wrote a book about Crowley's story, which was later made into the Hollywood film “Extraordinary Measures,” starring Harrison Ford and Brendan Fraser.

John Crowley, CEO of the Biotechnology Innovation Organization

Biotechnology Innovation Organization

Crowley has certainly made his mark in the biotech space. He helped build two biotech companies focused on rare diseases that were later acquired by larger pharmaceutical companies. Most recently, in December, BioMarin paid nearly $5 billion for Amicus Therapeutics, a company Crowley helped grow from a five-person startup in 2005 to a multibillion-dollar company when he left in 2024. Crowley left Amicus to become BIO's CEO.

Since taking that role, he has become increasingly outspoken about China's biotech sector, advocating for the United States to become more competitive.

“We need to reduce dependence on Chinese biotechnology,” Crowley said. “Once they are the dominant player, they will decide who gets what drugs and technologies.”

Crowley has seen firsthand the growth of Chinese biotechnology. “I go back to just 10 years ago, maybe, working in China. There were maybe a couple hundred real R&D biotech companies in China. By our calculations, today there are more than 4,000,” he said.

At the same time, Beijing is lowering regulatory hurdles for drugmakers conducting research in China, meaning treatments they are working on can enter clinical trials more quickly. This is attractive to both major drugmakers and smaller researchers around the world, who see drug development in China as a faster and cheaper option than in the United States. A recent article in STAT described the emergence of a Chinese incubator, ATLATL, highlighting how it has been able to develop relationships with clients that span the entire drug development process.

For Crowley, a former naval intelligence officer, the rise of biotechnology in China is a threat not only to the industry it represents, but also to the millions of patients who depend on rare disease research emerging from American universities.

“Our research is based on our great academic institutions [is] “It's a notable strategic advantage for the United States,” Crowley said. “Today it is threatened.”

“The biggest threat comes from China and the rise of Chinese biotechnology,” Crowley said.

“We cannot allow China to win in biotechnology,” he said.

Crowley is not alone with his concerns. Former FDA Commissioner Scott Gottlieb, a member of CNBC's Cures Advisory Board, dedicates a chapter of his upcoming book, “The Miracle Century,” to the rise of biotech in China. In the book, Gottlieb makes the case that as Beijing has streamlined the regulatory approval process so innovative medical treatments reach the market sooner, investment in those technologies has flowed from the United States to China. He wrote:

“If this drift continues and more drug discoveries migrate from the United States to China, we could see our capacity for innovation begin to erode. As capital flows to Chinese companies, American biotech hubs like Boston and San Francisco, long hotbeds of innovative science, may shrink. Restoring that American ecosystem would be anything but easy.”

The displacement of capital is not theoretical. It's happening.

A September paper published in Nature found that between 2020 and 2025, 11 of the largest pharmaceutical players committed more than $150 billion in deals to access assets developed in Asia, primarily China.

And data collected for another upcoming book, “Innovation is the Best Medicine,” by Dr. Roderick Wong, a physician and founder and managing partner of life sciences investment firm RTW Investments, shows that between 2013 and 2025, China tripled its share of global clinical trial starts.

Political think tanks and policymakers in the United States have taken notice.

In November, the nonpartisan Atlantic Council published an analysis that identified pharmaceuticals as China's next trade weapon, comparing the shift of biotech innovation to China with the offshoring of semiconductor chip manufacturing.

Spurred by concerns about corporate espionage, access to sensitive genetic data and memories of supply chain bottlenecks faced by the global medical supply industry after the Covid pandemic, Congress in late 2025 passed the Biosecurity Act, which President Donald Trump later signed into law as part of the massive $901 billion defense spending bill.

The Biosafety Act prohibits biotechnology companies that receive federal funds from doing business with companies that the United States designates as “biotechnology companies of concern.” While it will not ban all business that U.S. biotech companies do with China, and the law's language was watered down from an earlier version of the bill, the law is forcing some U.S.-based companies to reexamine their ties to China.

But for people living with a rare disease, the problem is not so clear. Rare diseases do not respect borders. And parents seeking life-saving treatment for their children don't care whether it comes from the United States or China. Innovation in the rare disease space is a good thing. And in a field where there may be only two or three experts in the world on a given disease, that innovation is often the result of international collaboration. Increasingly, this innovation comes from China.

It is an enigma that does not go unnoticed by Gottlieb, who recognized that Beijing's innovation is good for patients with rare diseases. At least in the short term. “However, if the end result is that the fragile American innovation sector is hollowed out and we lose our own innovation engine, that is bad,” Gottlieb said in a text. “The priority objectives of Chinese drug manufacturers may not reflect our priority objectives.”

“As China erodes other parts of our ecosystem, it could empty everything,” he added.

Both Gottlieb and Crowley said the real key to maintaining America's leadership in biotechnology is getting regulators to treat rare diseases, which might affect only a few hundred people, as opposed to those with larger patient populations. Rare disease researchers agree, arguing that a more streamlined approval process for rare disease treatments by the FDA would dramatically reduce the cost of bringing a new treatment to market in the US.

David Liu, a gene-editing pioneer whose lab at Harvard University and the Broad Institute is at the forefront of genetic research, said he has called on the FDA to take a more lenient stance when evaluating new treatments for rare diseases. One example Liu pointed to: current guidelines for cell and gene therapies that require a company to demonstrate three large-scale manufacturing processes before a treatment can gain final approval.

“Large-scale production typically costs $7 million for gene-editing treatments of rare genetic diseases,” Liu said. “Typically, a single production can already treat more patients than there are in the entire world. So you're just asking companies to waste an additional $14 million.”

Critics of current FDA policies argue that using a different set of standards for rare disease treatments would reduce development costs and help drugs reach people who need them more quickly, and could spur a new wave of investment in this space.

“Let's think creatively,” Crowley said. “Don't apply the same standards to a rare disease that affects 100 children as you do to a treatment designed for a disease that affects millions of people.”

“We need a system that works better,” he said.

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