China takes lead in CGT trials amid Western funding challenges

In 2019, the number of CGT trial initiations was similar between China and the US. However as the West has suffered funding complications, China has now become a leader in the space. Credit: Lichtgeschwindigkeit/Shutterstock.com

Despite widespread claims of a boom in the cell and gene therapy (CGT) landscape, analysis reveals that trial initiations have remained steady year over year – with China emerging as the global leader in CGT trials.

Biotechs have been tackling a whirlwind of challenges in the past few years, including financing, enrollment difficulties, and global political shifts causing regulatory changes, all of which have also impacted CGT companies.

CGT companies have faced complications beyond this, including safety concerns and manufacturing difficulties, which further complicate pipeline development.

According to GlobalData’s recent report, The State of the Biopharmaceutical Industry 2025 – Mid-Year Update, the global CGT market is estimated to be worth $79.3bn by 2030, with a 40% compound annual growth rate (CAGR). Oncology is predicted to continue as the top therapy area, followed by central nervous system (CNS) disorders and metabolic disorders. And as the number of applications for CGTs continues to grow, in the future, no indication is off the table, showing a real desire for the development of these kinds of drugs.

GlobalData is the parent company of Clinical Trials Arena.

GlobalData’s Pharmaceutical Intelligence Centre reports that the number of CGT trial initiations has remained relatively steady each year since 2019. A huge proportion of these trials were single-country, likely due to the feasibility of running multi-country CGT studies and the manufacturing burden of these kinds of therapies.

“There are regions where the number of trials is growing, like Asia and then the regions where the number of trials is going down, like Europe and the US. It in some ways makes sense that the number is more or less the same year to year,” says Vladimir Ivošev, senior business development director for Northway Biotech and commercial advisor to SyVento BioTech, a Polish contract development and manufacturing organisation (CDMO), who says this has been a trend in recent years.

GlobalData analysis supports this, showing a huge jump in the number of trials initiated in China from 2019 to 2024, with China having around double the number of CGT studies as the US in the year.

Miguel Forte, President-Elect and Board of Directors member of the International Society of Cell and Gene Therapy (ISCT), agrees that development in the East, specifically China, is higher than in the US, likely due to the country facing fewer challenges financially than the West.

“The expansion in China is faster, while the US is struggling more than we would like. Europe still has not solved the problem of being able to really expedite clinical trials. This is because of the challenging biotech environment, particularly in Europe and a bit in the US, but less so in China. As a result, we’re seeing limited growth behind the expectations,” Forte explains.

As Forte raises, financial barriers have been an issue for all biotechs since the 2021 boom. While this is an issue across all therapy areas, the high cost of developing and manufacturing CGTs means it has been particularly difficult for the sector to initiate new trials.

“The financial barrier is having an impact because we have more expensive clinical trials. If the regulators can somehow lower the cost and maybe adjust the requirements, that would be extremely beneficial for the space,” says Monika Paule, CEO of Caszyme, a biotech specialising in CRISPR therapies.

Not only has private financing been an issue, but in recent months, government-led grants have also been reduced, which could lead to a drop in the number of Phase I trial initiations in the coming years, Ivošev believes.

“This is a huge blow – to have very little or no governmental support makes things even harder. Private equity and venture funds are getting more reluctant to fund this kind of early-stage development,” Ivošev explains. “There are now severe cuts in the pipelines. Many companies had multiple projects and products in the pipeline, but now everything is more streamlined because they are more conscious about their cash runway.”

While funding remains scarce, the number of trial initiations will likely remain similar; however, those that do make it to the clinic will likely be those that are likely to be more impactful, says Jon Ellis, CEO of Trenchant Biosciences, a CAR-T manufacturing company.

“I imagine that the trials that are going to get funded are the ones that are really going to make a difference and offer something a bit unique. So hopefully it’ll work out. But right now, it’s still, there’s a lot of scrambling around trying to figure out what to do,” says Ellis.

One of the main reasons for trial terminations in all trials is low recruitment. Companies in the CGT space, including Sarepta and Pfizer, have suffered patient deaths in trials. For patients who have alternative options for their disease, this may make them cautious about enrolling on a CGT study, Ivošev believes, which could also impact the number of active CGT trials down the line.

“For diseases where there are basically no alternative therapies, it probably will not change much because people have no other option, and patients are willing to try anything. Some indications where there are alternative treatments that may be where people are not sure whether they want this kind of investigative and permanent change,” Ivošev says.

However, given the number of patients who do benefit from these studies, Paule hopes education may be the answer to patients’ fear of these kinds of modalities.

Given the back and forth with Sarepta, as well as other gene therapy clinical holds such as Rocket’s Danon Disease therapy hold, it is clear the FDA are being scrutinous with safety data, says Ellis. While this is impacting current trials, it will likely make biotechs more cautious before bringing a drug to first-in-human studies, he adds, making sure the safety profile is as well established as possible before human dosing, which will help avoid trial terminations due to safety in the future.

With the back and forth in recent months between the US Food and Drug Administration (FDA) and gene therapy developer Sarepta, there are questions as to whether, with Vinay Prasad at the helm of the CDER, this will impact trial initiations going forward.

“It’s probably not the best signal for gene therapy overall. There will probably be some headwinds, now that he’s back in at the FDA,” says Ivošev.

However, Paule believes his return is unlikely to impact clinical trial initiations in the coming years and hopes that growing evidence around the therapies will also spark regulator confidence in the modalities.

“We have a very robust preclinical space; there are around 1500 trials in the space, and that’s a lot of data, which eventually will fill in the regulatory organisations with more trust in the field,” Paule adds.

Given the challenges facing Western biotechs, growth in the CGT landscape is expected to be gradual. However, as cell and gene therapies become more integrated into mainstream treatment, their safety profiles improve, and manufacturing becomes more accessible and cost-effective, Forte anticipates a rise in trial initiations. While this evolution may take several years, the sector is well-positioned to experience significant growth in the not-too-distant future.

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