Highlights
Quote
EsoBiotec worked with Chinese doctors to run an investigator-initiated trial, known in the industry as an IIT. Unlike a traditional clinical trial that requires approval from national regulators, researchers just need clearance from the Chinese research hospital running the study to start an IIT.
Quote
IITs have grown increasingly popular among China researchers over the past decade, and have helped China’s fast-growing biotech sector catch up with rivals in the US and Europe.
Quote
While the small trials can’t be used for approval, they offer a nimble way to let companies see whether their medicines are promising — and help them attract new backing.
Quote
According to one analysis conducted by Beijing-based research center Changping Laboratory, the number of Chinese IITs for cell and gene therapies grew 11-fold between 2015 and early 2024, totaling more than 1,000 studies
Quote
While biopharma companies have long conducted trials around the world, including in China, IITs have emerged as a new tactic.
Quote
many US and Chinese drugmakers alike told Endpoints they are at least considering, if not already planning or currently running, an IIT in China.
Quote
“Everybody in biotech is reevaluating how they work with China,”
Quote
“Their model enables a much faster path to clinical validation, and for a startup, the most important thing you need is clinical validation. You’d have to be crazy not to be paying attention.”
Quote
And he expects more US biotechs to turn to China for IITs: “If they’re not doing it, they’re going to lose.”
Quote
Those early data paid off. Johnson & Johnson struck a partnership with Legend later that year. Their cell therapy Carvykti surpassed $1 billion in sales in 2025.
Quote
For EsoBiotec, the advantages outweighed the challenges thanks to the most important feature of IITs in China: their regulatory flexibility. While physicians can run IITs in the US, they still need the FDA’s approval. In China, permission is granted by local authorities, such as ethics and science committees at research hospitals, which can make reviews faster and less stringent.
Quote
Another advantage is looser manufacturing requirements.
Quote
But in IITs, companies can make smaller batches that are “GMP-like” or “halfway to GMP,” which saves time and money, several biotech leaders told Endpoints.
Quote
Lu Han, CEO of Imviva Biotech (formerly known as Bioheng Therapeutics), said IITs can help a company “fail fast.”
Quote
“If you really want to crack this code of immune rejection, you might need a little more than just one or two trials – maybe 10 or even more,”
Quote
“When the developers are looking outside our country for early-stage clinical work, we need to pick up our game,”
Quote
There are also lingering concerns around rigor and data trustworthiness. But those are no longer the roadblock they once were. Some experts believe China’s newly implemented standards for IITs could improve perceptions and scrupulousness of the studies.
Quote
“We’re going to need to replicate that paradigm here, or something like it, because there’s immense power in it,” Nuwaysir said. “There’s a lot for us to learn from what they’re doing. And frankly, if we don’t learn, there’s going to be more pain coming for biotech.”
Clean Copy
China has a cheap, quick and quiet way to test novel therapies. Western genetic medicine makers want in
Earlier this year, the Belgian cell therapy startup EsoBiotec had only 12 employees and had raised €22 million to develop a cutting-edge infusion that turns a patient’s immune cells into destroyers of disease.
But despite its meager resources, EsoBiotec managed to do something that its better-capitalized peers hadn’t: It became the first to report clinical data on a highly-anticipated cancer treatment called an in vivo CAR-T therapy.
And it did so with help from China.
EsoBiotec worked with Chinese doctors to run an investigator-initiated trial, known in the industry as an IIT. Unlike a traditional clinical trial that requires approval from national regulators, researchers just need clearance from the Chinese research hospital running the study to start an IIT. EsoBiotec treated its first multiple myeloma patient in November and quickly showed that its therapy could keep the blood cancer at bay. Just months later, AstraZeneca said it planned to buy the company for $425 million.
Mark Cobbold
“We’re all comfortable with the US being the dominant space for drug discovery and drug development, but I think that is definitely shifting,” AstraZeneca’s head of immuno-oncology and cell therapy Mark Cobbold told Endpoints News.
IITs have grown increasingly popular among China researchers over the past decade, and have helped China’s fast-growing biotech sector catch up with rivals in the US and Europe. The very leniency that once made American and European companies view these Chinese studies with skepticism is now providing them a unique opportunity to cheaply and quickly assess their experimental medicines.
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
Compared with past years, does 2026 feel like a time when biopharma should take risks or be conservative?
Select one
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
What region do you think will be the most important hub for biopharma research in 2030?
Select one
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
What do you think will best describe M&A activity in 2026?
Select one
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
How would you describe the industry mood this week compared to last year?
Select one
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
In your own words, how would you describe the biggest challenges and opportunities facing biopharma in 2026? (optional)
Open-ended
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
Are you attending J.P. Morgan in person this year?
Select one
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
Would you like the final poll analysis sent to you by email?
Select one
Please enter a valid email address
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
Thank you for taking the survey!
We appreciate your time and expert feedback.
Do you have an idea for a future Pulse Survey?
Drop us a note
Want to host your own survey here?
Contact Endpoints Signal
Thank you for taking the survey!
We appreciate your time and expert feedback.
Do you have an idea for a future Pulse Survey?
Drop us a note
Want to host your own survey here?
Contact Endpoints Signal
While the small trials can’t be used for approval, they offer a nimble way to let companies see whether their medicines are promising — and help them attract new backing. That’s especially important for cell and gene therapy developers, where Chinese companies are beginning to pull even or ahead of Western biotech, prompting US policymakers to look at changes.
According to one analysis conducted by Beijing-based research center Changping Laboratory, the number of Chinese IITs for cell and gene therapies grew 11-fold between 2015 and early 2024, totaling more than 1,000 studies — though other data sources suggest the number may be even higher. Because trials are local and there’s no centralized database to monitor them, it can be hard to know exactly how widespread they are. It’s also unknown how many of these studies have ties to companies outside of China.
While biopharma companies have long conducted trials around the world, including in China, IITs have emerged as a new tactic. In conversations with more than a dozen biotech leaders working on cutting-edge cell and gene therapies, many US and Chinese drugmakers alike told Endpoints they are at least considering, if not already planning or currently running, an IIT in China.
Emile Nuwaysir
“Everybody in biotech is reevaluating how they work with China,” said Emile Nuwaysir, CEO of the recently launched Cambridge, MA-based genetic medicine startup Stylus Medicine. “Their model enables a much faster path to clinical validation, and for a startup, the most important thing you need is clinical validation. You’d have to be crazy not to be paying attention.”
Nuwaysir said that Stylus is considering a China IIT for its first clinical trial, although he declined to share the company’s specific plans.
And he expects more US biotechs to turn to China for IITs: “If they’re not doing it, they’re going to lose.”
The appeal of the IIT
IITs played a major part in how China became a competitive force in cell and gene therapy. Legend Biotech, for instance, presented astonishing results in 2017 from an IIT study of its multiple myeloma CAR-T cell therapy.
Those early data paid off. Johnson & Johnson struck a partnership with Legend later that year. Their cell therapy Carvykti surpassed $1 billion in sales in 2025.
IITs must be run by researchers at a hospital or academic center. Close-knit relationships between Chinese biotech companies and physicians at top hospitals are key to the profluence of IITs and a high hurdle for Western drugmakers looking to break in. To run its IIT, EsoBiotec worked with Shenzhen-based Pregene Biopharma, which worked with two hospitals in Wuhan.
For EsoBiotec, the advantages outweighed the challenges thanks to the most important feature of IITs in China: their regulatory flexibility. While physicians can run IITs in the US, they still need the FDA’s approval. In China, permission is granted by local authorities, such as ethics and science committees at research hospitals, which can make reviews faster and less stringent.
JP Latere
“We gained probably two years,” EsoBiotec CEO JP Latere said.
Another advantage is looser manufacturing requirements. In Phase 1 studies, companies have to adhere to Good Manufacturing Practice (GMP) standards, which require intense quality control and heavy documentation to ensure the process is replicable. But in IITs, companies can make smaller batches that are “GMP-like” or “halfway to GMP,” which saves time and money, several biotech leaders told Endpoints.
And while drug regulators often require companies to monitor patients for long periods before ramping up to higher doses, the turnaround is shorter in an IIT. Companies work with the hospital directly to modify dosing, the study design and even the drug itself.
Lu Han, CEO of Imviva Biotech (formerly known as Bioheng Therapeutics), said IITs can help a company “fail fast.” His company is using IITs to develop off-the-shelf cell therapies, an area where many US companies have struggled.
“If you really want to crack this code of immune rejection, you might need a little more than just one or two trials – maybe 10 or even more,” Han said.
US contends with rising IIT competition
New FDA leaders have acknowledged that early-stage work in the US needs to speed up. Lawmakers, too, are paying attention.
“When the developers are looking outside our country for early-stage clinical work, we need to pick up our game,” Sen. Bill Cassidy (R-LA) said at a hearing on US biotech competitiveness.
Some US biotech investors have called for the FDA to follow China’s model, allowing for decentralized approvals for first-in-man trials.
Instead, many are going to China, where they can act now. Umoja Biopharma, one of the most well-funded in vivo CAR-T companies, is conducting its first clinical test in a China IIT. Gilead has also struck a deal with China’s Pregene to study cell therapies in human trials more quickly. Some new startups have told Endpoints they are banking on IITs to get their first clinical data with a modest amount of funding.
Even so, FDA Commissioner Marty Makary has expressed skepticism about trials conducted in China. He recently suggested that companies with early-stage studies outside the US might have to pay a higher user fee.
There are also lingering concerns around rigor and data trustworthiness. But those are no longer the roadblock they once were. Some experts believe China’s newly implemented standards for IITs could improve perceptions and scrupulousness of the studies. Many genetic medicine companies have already been going to countries like Australia and New Zealand to conduct their Phase 1 studies. Tests in China are just the next step.
Nuwaysir, the Stylus CEO, said the US would benefit from “the elements of nimbleness” that China provides.
“We’re going to need to replicate that paradigm here, or something like it, because there’s immense power in it,” Nuwaysir said. “There’s a lot for us to learn from what they’re doing. And frankly, if we don’t learn, there’s going to be more pain coming for biotech.”
Annotated Copy
China has a cheap, quick and quiet way to test novel therapies. Western genetic medicine makers want in
Earlier this year, the Belgian cell therapy startup EsoBiotec had only 12 employees and had raised €22 million to develop a cutting-edge infusion that turns a patient’s immune cells into destroyers of disease.
But despite its meager resources, EsoBiotec managed to do something that its better-capitalized peers hadn’t: It became the first to report clinical data on a highly-anticipated cancer treatment called an in vivo CAR-T therapy.
And it did so with help from China.
EsoBiotec worked with Chinese doctors to run an investigator-initiated trial, known in the industry as an IIT. Unlike a traditional clinical trial that requires approval from national regulators, researchers just need clearance from the Chinese research hospital running the study to start an IIT. EsoBiotec treated its first multiple myeloma patient in November and quickly showed that its therapy could keep the blood cancer at bay. Just months later, AstraZeneca said it planned to buy the company for $425 million.
Mark Cobbold
“We’re all comfortable with the US being the dominant space for drug discovery and drug development, but I think that is definitely shifting,” AstraZeneca’s head of immuno-oncology and cell therapy Mark Cobbold told Endpoints News.
IITs have grown increasingly popular among China researchers over the past decade, and have helped China’s fast-growing biotech sector catch up with rivals in the US and Europe. The very leniency that once made American and European companies view these Chinese studies with skepticism is now providing them a unique opportunity to cheaply and quickly assess their experimental medicines.
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
Compared with past years, does 2026 feel like a time when biopharma should take risks or be conservative?
Select one
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
What region do you think will be the most important hub for biopharma research in 2030?
Select one
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
What do you think will best describe M&A activity in 2026?
Select one
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
How would you describe the industry mood this week compared to last year?
Select one
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
In your own words, how would you describe the biggest challenges and opportunities facing biopharma in 2026? (optional)
Open-ended
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
Are you attending J.P. Morgan in person this year?
Select one
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
Would you like the final poll analysis sent to you by email?
Select one
Please enter a valid email address
Hello Reader,
Endpoints is fielding its next Pulse Poll — a one-minute check-in on the biopharma mood around JPM 2026.
You don’t need to attend the conference to take the poll. Results are confidential.
Thank you for taking the survey!
We appreciate your time and expert feedback.
Do you have an idea for a future Pulse Survey?
Drop us a note
Want to host your own survey here?
Contact Endpoints Signal
Thank you for taking the survey!
We appreciate your time and expert feedback.
Do you have an idea for a future Pulse Survey?
Drop us a note
Want to host your own survey here?
Contact Endpoints Signal
While the small trials can’t be used for approval, they offer a nimble way to let companies see whether their medicines are promising — and help them attract new backing. That’s especially important for cell and gene therapy developers, where Chinese companies are beginning to pull even or ahead of Western biotech, prompting US policymakers to look at changes.
According to one analysis conducted by Beijing-based research center Changping Laboratory, the number of Chinese IITs for cell and gene therapies grew 11-fold between 2015 and early 2024, totaling more than 1,000 studies — though other data sources suggest the number may be even higher. Because trials are local and there’s no centralized database to monitor them, it can be hard to know exactly how widespread they are. It’s also unknown how many of these studies have ties to companies outside of China.
While biopharma companies have long conducted trials around the world, including in China, IITs have emerged as a new tactic. In conversations with more than a dozen biotech leaders working on cutting-edge cell and gene therapies, many US and Chinese drugmakers alike told Endpoints they are at least considering, if not already planning or currently running, an IIT in China.
Emile Nuwaysir
“Everybody in biotech is reevaluating how they work with China,” said Emile Nuwaysir, CEO of the recently launched Cambridge, MA-based genetic medicine startup Stylus Medicine. “Their model enables a much faster path to clinical validation, and for a startup, the most important thing you need is clinical validation. You’d have to be crazy not to be paying attention.”
Nuwaysir said that Stylus is considering a China IIT for its first clinical trial, although he declined to share the company’s specific plans.
And he expects more US biotechs to turn to China for IITs: “If they’re not doing it, they’re going to lose.”
The appeal of the IIT
IITs played a major part in how China became a competitive force in cell and gene therapy. Legend Biotech, for instance, presented astonishing results in 2017 from an IIT study of its multiple myeloma CAR-T cell therapy.
Those early data paid off. Johnson & Johnson struck a partnership with Legend later that year. Their cell therapy Carvykti surpassed $1 billion in sales in 2025.
IITs must be run by researchers at a hospital or academic center. Close-knit relationships between Chinese biotech companies and physicians at top hospitals are key to the profluence of IITs and a high hurdle for Western drugmakers looking to break in. To run its IIT, EsoBiotec worked with Shenzhen-based Pregene Biopharma, which worked with two hospitals in Wuhan.
For EsoBiotec, the advantages outweighed the challenges thanks to the most important feature of IITs in China: their regulatory flexibility. While physicians can run IITs in the US, they still need the FDA’s approval. In China, permission is granted by local authorities, such as ethics and science committees at research hospitals, which can make reviews faster and less stringent.
JP Latere
“We gained probably two years,” EsoBiotec CEO JP Latere said.
Another advantage is looser manufacturing requirements. In Phase 1 studies, companies have to adhere to Good Manufacturing Practice (GMP) standards, which require intense quality control and heavy documentation to ensure the process is replicable. But in IITs, companies can make smaller batches that are “GMP-like” or “halfway to GMP,” which saves time and money, several biotech leaders told Endpoints.
And while drug regulators often require companies to monitor patients for long periods before ramping up to higher doses, the turnaround is shorter in an IIT. Companies work with the hospital directly to modify dosing, the study design and even the drug itself.
Lu Han, CEO of Imviva Biotech (formerly known as Bioheng Therapeutics), said IITs can help a company “fail fast.” His company is using IITs to develop off-the-shelf cell therapies, an area where many US companies have struggled.
“If you really want to crack this code of immune rejection, you might need a little more than just one or two trials – maybe 10 or even more,” Han said.
US contends with rising IIT competition
New FDA leaders have acknowledged that early-stage work in the US needs to speed up. Lawmakers, too, are paying attention.
“When the developers are looking outside our country for early-stage clinical work, we need to pick up our game,” Sen. Bill Cassidy (R-LA) said at a hearing on US biotech competitiveness.
Some US biotech investors have called for the FDA to follow China’s model, allowing for decentralized approvals for first-in-man trials.
Instead, many are going to China, where they can act now. Umoja Biopharma, one of the most well-funded in vivo CAR-T companies, is conducting its first clinical test in a China IIT. Gilead has also struck a deal with China’s Pregene to study cell therapies in human trials more quickly. Some new startups have told Endpoints they are banking on IITs to get their first clinical data with a modest amount of funding.
Even so, FDA Commissioner Marty Makary has expressed skepticism about trials conducted in China. He recently suggested that companies with early-stage studies outside the US might have to pay a higher user fee.
There are also lingering concerns around rigor and data trustworthiness. But those are no longer the roadblock they once were. Some experts believe China’s newly implemented standards for IITs could improve perceptions and scrupulousness of the studies. Many genetic medicine companies have already been going to countries like Australia and New Zealand to conduct their Phase 1 studies. Tests in China are just the next step.
Nuwaysir, the Stylus CEO, said the US would benefit from “the elements of nimbleness” that China provides.
“We’re going to need to replicate that paradigm here, or something like it, because there’s immense power in it,” Nuwaysir said. “There’s a lot for us to learn from what they’re doing. And frankly, if we don’t learn, there’s going to be more pain coming for biotech.”