If US biotechnology gives China a head start, it could help patients with rare diseases: experts
The growth of China’s biotechnology sector is breathtaking. 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 US biotech industry and also affects patients with rare diseases awaiting a cure.
Among the experts speaking out against China’s growing influence in the biotechnology sector is John Crowley, CEO 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 learned that his two young children had Pompe disease, a fatal genetic disorder, Crowley quit his job in marketing to search for a cure. He teamed up with a researcher working on 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 later became the Hollywood film Extraordinary Measures starring Harrison Ford and Brendan Fraser.
John Crowley, CEO of the Biotechnology Innovation Organization
Biotechnological innovation organization
Crowley has undoubtedly made a name for himself 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 that helped Crowley grow from a five-person startup in 2005 to a multibillion-dollar company when he exited in 2024. Crowley left Amicus to become CEO of BIO.
Since taking office, he has become increasingly vocal about the Chinese biotech sector and advocates for the United States to become more competitive.
“We need to reduce dependence on Chinese biotech companies,” Crowley said. “Once they are the dominant player, they will decide who gets what drugs and technologies.”
Crowley has witnessed the growth of Chinese biotechnology firsthand. “I remember about ten years ago when I was working in China. There were maybe a few hundred real R&D biotech companies in China. By our count, there are now over 4,000,” he said.
At the same time, Beijing is reducing regulatory hurdles for drugmakers conducting research in China, allowing the treatments they are working on to move into clinical trials more quickly. This is attractive to both large drugmakers and smaller researchers from around the world, for whom developing drugs in China is a faster and cheaper option than in the United States. A recent article in STAT detailed the rise of a Chinese incubator, ATLATL, and highlighted how it has managed to build relationships with customers across the drug development pipeline.
For Crowley, a former Navy intelligence officer, China’s rise in biotechnology poses a threat not only to the industry he represents, but also to the millions of patients who rely on rare disease research at American universities.
“Our research is based on our great academic institutions [is] “A remarkable 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 in his concerns. Former FDA Commissioner Scott Gottlieb, a member of the CNBC Cures Advisory Board, devotes a chapter to China’s rise in biotechnology in his upcoming book “The Miracle Century.” In the book, Gottlieb argues that investment in these technologies has flowed from the United States to China as Beijing has simplified the regulatory approval process to allow breakthrough medical treatments to come to market more quickly. He wrote:
“If this trend continues and more drug research moves from the U.S. to China, we could see our ability to innovate begin to wane. As capital flows toward Chinese companies, U.S. biotechnology hubs like Boston and San Francisco, long the seeds of groundbreaking science, could shrink. Restoring this American ecosystem would be anything but easy.”
The movement of capital is not theoretical. It happens.
An article published in Nature in September found that 11 of the largest pharmaceutical companies struck more than $150 billion in deals between 2020 and 2025 to gain access to assets developed in Asia, primarily China.
And for another upcoming book “Innovation is the best medicine” by Dr. Data collected by Roderick Wong, a physician and founder and managing partner of life sciences investment firm RTW Investments, shows that China tripled its share of clinical trials initiated worldwide from 2013 to 2025.
Policy think tanks and lawmakers in the US have taken note.
In November, the nonpartisan Atlantic Council released an analysis identifying pharmaceuticals as China’s next trade weapon and comparing the relocation of biotechnology innovation to China to the relocation of semiconductor chip manufacturing abroad.
Prompted by concerns about corporate espionage, access to sensitive genetic data and reminders of the supply chain bottlenecks facing the global medical supply industry in the wake of the Covid pandemic, Congress passed the Biosecure Act in late 2025, which President Donald Trump later signed into law as part of the massive $901 billion defense spending bill.
The Biosecure Act prohibits biotech companies that receive federal funding from doing business with companies designated by the U.S. as “biotech companies of concern.” While it will not ban all American biotech companies’ dealings with China, and the wording of the law has been weakened from an earlier version of the bill, the law will force some U.S.-based companies to reconsider their relationships with China.
However, for people with a rare disease, the problem is not so clear-cut. Rare diseases know no borders. And parents seeking life-saving treatment for their child don’t care whether they come from the United States or China. Innovation in rare diseases is a good thing. And in a field where there may only be two or three experts in the world for a particular disease, this innovation is often the result of international collaboration. These innovations are increasingly coming from China.
This conundrum was not lost on Gottlieb, who acknowledged 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 U.S. innovation sector is hollowed out and we lose our own innovation engine, that is bad,” Gottlieb said in a text. “The primary goals of Chinese drugmakers may not reflect our primary goals.”
“If China hollows out other parts of our ecosystem, it could hollow out everything,” he added.
Both Gottlieb and Crowley said the real key to maintaining U.S. leadership in biotech is getting regulators to treat rare diseases that may affect only a few hundred people differently than those with larger patient populations. Rare disease researchers agree, arguing that a streamlined FDA approval process for rare disease treatments would dramatically reduce the cost of bringing a new treatment to market in the United States
David Liu, a gene editing pioneer whose lab at Harvard University and the Broad Institute is at the cutting edge of genetic research, said he has asked the FDA to take a more lenient stance in evaluating new treatments for rare diseases. One example Liu pointed to: Current guidelines for cell and gene therapies require a company to have three full production runs before a treatment can receive final approval.
“A full production run for gene editing treatments for rare genetic diseases typically costs $7 million,” Liu said. “One production run can typically treat more patients than there are in the world, so you’re just asking companies to throw away an additional $14 million.”
Critics of current FDA guidelines argue that using different standards for treating rare diseases would reduce development costs and help drugs get to the people who need them more quickly, and that it could spark a new wave of investment in the area.
“Let’s think creatively,” Crowley said. “Don’t apply the same standards for a rare disease affecting 100 children to a treatment developed for a disease affecting millions of people.”
“We need a system that works better,” he said.
