Keeping U.S. biotech ahead of China could aid rare disease patients: Experts

The growth of China’s biotechnology sector is astonishing. Beijing is pumping money into the sector, supporting research efforts and helping launch a new wave of laboratories and incubators in the country. This is a problem for the US biotech industry and also affects rare disease patients awaiting treatment.

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 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 fatal genetic disorder, Crowley quit his job in marketing to find a cure. He partnered with a researcher working on Pompe and founded a company that developed a treatment that would eventually 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; This book later became the Hollywood movie “Extraordinary Measures” starring Harrison Ford and Brendan Fraser.

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

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

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

Crowley saw the growth of China’s biotechnology firsthand. “I think back maybe just 10 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 over 4,000 companies today,” he said.

At the same time, Beijing is reducing regulatory barriers to drugmakers conducting research in China; This means the treatments they are working on can enter clinical trials faster. This is attractive to both large drug manufacturers and small researchers around the world, who see drug development in China as a faster and cheaper option than in the United States. A. Latest article in STAT He profiled the rise of ATLATL, a Chinese incubator, highlighting how it was able to foster relationships with customers spanning the entire drug development pipeline.

According to Crowley, a former naval intelligence officer, China’s rise in biotechnology poses a threat not only to the industry it represents but also to the millions of patients who rely on rare disease research coming out of American universities.

“Our research is based in our major academic institutions [is] “It’s a significant strategic advantage for the United States,” Crowley said. “It is under threat today.”

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

“We can’t let China 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 finds that investment in these technologies is flowing from the United States to China as Beijing simplifies the regulatory approval process, allowing groundbreaking medical treatments to come to market more quickly. He wrote:

The change in capital is not theoretical. This is happening.

An article published in September Nature It was revealed that between 2020 and 2025, 11 of the largest pharmaceutical companies have committed more than $150 billion in deals to access assets developed in Asia, especially China.

Physician Dr., founder and managing partner of life sciences investment company RTW Investments. Data collected for Roderick Wong’s upcoming book “Innovation is the Best Medicine” shows that China tripled its share of global clinical trial initiatives between 2013 and 2025.

Political think tanks and lawmakers in the US took notice.

In November, the nonpartisan Atlantic Council published an analysis that described pharmaceuticals as: China’s next trading weaponHe likens the shift of innovation in biotechnology to China to shifting semiconductor chip production abroad.

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

The Biosecurity Act prohibits biotechnology companies that receive federal funding from doing business with companies that the United States designates as “related biotechnology companies.” Although it does not ban all business by U.S. biotech companies with China and the language of the bill has been softened compared to the previous version of the bill, the law does put pressure on China. Some US-based companies will reconsider their ties with China.

But for people living with a rare disease, the problem is not so clear-cut. Rare diseases do not respect borders. And parents seeking a life-saving treatment for their children 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 may be only two or three experts in the world on any given disease, this innovation is often the result of international collaboration. This innovation is coming more and more often from China.

It’s a conundrum that doesn’t escape Gottlieb, who concedes that Beijing’s innovation is good for rare disease patients. At least in the short term. “But it would be bad if the result is that the fragile US innovation sector is gutted and we lose our own innovation engine,” Gottlieb said in a text. “Chinese drug manufacturers’ priority goals may not reflect our priority goals.”

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

Both Gottlieb and Crowley said the real key to maintaining U.S. leadership in biotechnology is to ensure regulators treat rare diseases that may affect only a few hundred people, as opposed to those with larger patient populations. Rare disease researchers agree, arguing that 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 cutting edge of genetic research, said he wants 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 full-scale production studies before a treatment can receive final approval.

“The cost of a full-scale production run for rare genetic disease gene editing treatments is typically $7 million,” Liu said. “A production run could normally treat more patients than are available globally. So you’re asking companies to throw away an extra $14 million.”

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

“Let’s think creatively,” Crowley said. “Don’t apply the same standards for a rare disease in which there are 100 children to a treatment designed for a disease in which there are millions of people.”

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