Keeping U.S. biotech ahead of China could help rare disease patients, experts say

The growth of China’s biotechnology sector has been astounding. Beijing is pumping money into the industry, supporting research efforts and helping launch a new wave of labs and incubators in the country. It’s a problem for the U.S. biotech industry and it also affects rare disease patients who are waiting for a cure.

Among the experts speaking out against China’s growing influence in the biotech sector is John Crowley, CEO of the lobbying group Biotechnology Innovation Organization, or 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 try to find a cure. He teamed up with a researcher who worked on Pompe and created a company that ultimately developed a treatment to save the lives of his children and thousands of others.

If that 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.

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

Since taking office, he has become increasingly outspoken about China’s biotechnology sector, advocating for the United States to become more competitive.

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

Crowley witnessed the growth of Chinese biotechnology. “Just 10 years ago, I was working in China. There were maybe a few hundred real biotechnology R&D companies in China. By our calculations, there are more than 4,000 today,” he said.

At the same time, Beijing is reducing the regulatory hurdles faced by drugmakers conducting research in China, meaning the treatments they are working on can move into clinical trials more quickly. This attracts both large drugmakers and small researchers around the world, who see drug development in China as a faster and less expensive option than in the United States. A recent article in STAT described the rise of a Chinese incubator, ATLATL, highlighting how it was able to develop relationships with clients spanning the entire drug development pipeline.

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

“Our research, anchored in our great academic institutions, constitutes a remarkable strategic advantage for the United States,” Crowley said. “It’s under threat today.”

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

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

Crowley isn’t the only one with his concerns. Former FDA Commissioner Scott Gottlieb, a member of the CNBC Cures Advisory Board, devotes a chapter of his upcoming book, “The Miracle Century,” to the rise of biotechnology in China. In the book, Gottlieb lays out the fact that as Beijing has streamlined the regulatory approval process so that breakthrough medical treatments can come to market more quickly, investment in these technologies has flowed from the United States to China. He wrote:

The movement of capital is not theoretical. It happens.

An article published in September in Nature reveals that between 2020 and 2025, 11 of the largest pharmaceutical players have committed more than $150 billion in transactions to access assets developed in Asia, mainly in China.

And data collected for another forthcoming 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 clinical trial launches worldwide.

Policy think tanks and U.S. lawmakers have taken note.

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

Driven by concerns about industrial espionage, access to sensitive genetic data and memories of the supply chain bottlenecks the global medical supplies industry faced after the Covid pandemic, Congress passed the Biosecurity 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 biotechnology companies that receive federal funds from doing business with companies that the United States designates as “biotechnology companies of concern.” While it does not ban all U.S. biotech companies’ activities with China and the law’s language has been relaxed from an earlier version of the bill, the law requires some U.S.-based companies to reexamine their ties to China.

But for people living with a rare disease, the problem is not as obvious. Rare diseases do not respect borders. And parents seeking life-saving treatment for their child don’t care whether it comes from the United States or China. Innovation in the field of rare diseases is a good thing. And in a field where there are only two or three experts in the world for a given disease, this innovation is often the result of international collaboration. More and more often, this innovation comes from China.

It’s a conundrum not lost on Gottlieb, who acknowledged that Beijing’s innovation is beneficial for rare disease patients. At least in the short term. “If the end result is that America’s fragile innovation sector becomes hollow and we lose our own innovation engine, that’s bad,” Gottlieb said in a text. “Chinese drugmakers’ priority targets may not reflect our priority targets.”

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

Gottlieb and Crowley said the real key to maintaining U.S. leadership in biotechnology is getting regulators to address rare diseases, which might affect only a few hundred people, unlike those with larger patient populations. Rare disease researchers agree, saying a more streamlined FDA approval process for rare disease treatments would significantly 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 forefront of genetic research, said he has asked the FDA to take a more lenient stance when evaluating new treatments for rare diseases. One example Liu cites: current guidelines for cell and gene therapies that require a company to demonstrate three rounds of large-scale manufacturing before a treatment can gain final approval.

“Large-scale manufacturing typically costs $7 million for gene-editing treatments for rare genetic diseases,” Liu said. “A single production usually already treats more patients than there are in the entire world. So you’re just asking companies to waste another $14 million.”

Critics of the FDA’s current policies argue that using a different set of standards for rare disease treatments would reduce development costs and help drugs get to people who need them more quickly, and that it could spur 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 designed for a disease affecting millions.”

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