On July 22, 2026, in Shanghai, the first National New Drug Global Conference "ATMP and CGT Going Global Special Session" was held by Tong Xieyi.

This year is a milestone for the Chinese cell gene therapy industry, bridging the past and ushering in the future.

Order No. 818 officially came into effect on May 1st, opening up an institutional channel for clinical research initiated by researchers to lead to clinical translation; At the same time, the global CGT competition landscape is accelerating its reshuffle, with 41 advanced therapeutic products already on the market in the United States and 30 in Europe, while only half of the 14 approved in China truly possess commercial vitality.

More importantly, after nearly a decade of explosive growth, the global share of CGT clinical research in China has shown signs of slowing growth for the first time in the past two years - funding bottlenecks, regulatory vacuum, and poor conversion pathways have begun to substantially constrain industry development.

Against this backdrop, Professor Ma Jun, the director of Harbin Institute of Hematology and Oncology, stepped onto the podium.

This 70 year old doctor is one of the few people who can cover the history of blood tumor treatment in China from beginning to end. From the early 1970s when the First Affiliated Hospital of Harbin Medical University treated patients with "713 Injection" (Cancer Spirit No.1), to participating in the evaluation of almost all Car-T, gene therapy, and stem cell therapies. For over half a century, he has been both a witness and a participant.

Professor Ma Jun: Director of Harbin Institute of Hematology and Oncology

01. Hematological tumors, leading the entire history of cancer prevention

The lymphatic system has entered a new era with malignant tumors, starting from the blood, "said Ma Jun.

In 1942, two pharmacologists Goodman and Gilman began using nitrogen mustard to treat Hodgkin's disease (published in 1943). Five years later, Dr. Farber from Boston Children's Hospital used methotrexate to relieve leukemia in children. This is the beginning of the era of chemotherapy.

In 1960, Professor Norville of Philadelphia Children's Hospital discovered a chromosomal translocation of chromosome 9 and 22 in a chronic myeloid leukemia patient in the outpatient department - the first tumor specific genetic marker in human history. Without this symbol, there would be no today and tomorrow of our current cancer treatment, "Ma Jun commented

Unfortunately, Professor Norville passed away unexpectedly in 2013 and was not awarded the Nobel Prize.

In 1996, Professor Drucker from Stanford presented an orange colored small molecule drug at an academic conference that could cause the disappearance of the Philadelphia chromosome. Five years later, imatinib was rapidly approved in the United States and entered China without even conducting any local clinical trials. Exemption from clinical trials entering China.

In the past, patients with chronic myeloid leukemia could only live for an average of three years, but now 88% of patients can achieve long-term disease-free survival, "said Ma Jun. This is the most impressive achievement of small molecule inhibitor targeted drugs.

In 2012, Professor Carl Juhn's team from the University of Pennsylvania successfully treated a 6-year-old girl with relapsed/refractory acute lymphoblastic leukemia using CAR-T therapy for the first time in the United States.

The girl's name is Emily, and she only has two months left to survive. After six days of cell transfusion, a miracle occurred. She fully recovered after one month, "Ma Jun mentioned Emily's three sentences at the ASCO conference -" My parents gave me my first life, medical staff gave me a second life, and the US government provided me with free medical assistance

I think the purpose of our party and government is also to serve the people, everything for the sake of the patients. In the near future, free medical care will also be provided for cancer patients.

Ma Jun called for CAR-T products to directly enter Class B medical insurance instead of Class C: "Only by entering Class B medical insurance can patients afford it

02. China has original content, but not enough

When it comes to original innovation, Ma Jun said that China's new drug research and development has gone through three stages: from the 1950s to the 1990s, it was tracking imitation; Next is the imitative innovation of me too, me better, and fast follow; We are now moving towards first in class.

Specifically, original innovation can be divided into eight dimensions: new targets, new mechanisms, new technologies, new structures, new indications, new dosage forms, new characteristics, and advanced treatment methods. Each item is not easy.

Does China have the ability to engage in primitive innovation? "Ma Jun asked himself and answered," Of course it does
He gave three examples. Tu Youyou's artemisinin is globally recognized. Shanghai Institute of Materia Medica's dimercaptosuccinic acid, a detoxifying drug for heavy metal poisoning, was used to save more than 3000 people after a train accident in the United States. Original from China. There is also arsenic trioxide in Harbin - the "713 Injection" purified from arsenic by pharmacist Han Taiyun in 1971, which was later proven to cure acute promyelocytic leukemia. The Memorial Sloan Kettering Cancer Center in New York, USA, used the Harbin Protocol to treat 9 relapsed and refractory acute promyelocytic leukemia patients and achieved complete remission, shocking the world. China and the United States quickly approved arsenic trioxide injection for the treatment of APL.

Pharmacist Han Taiyun passed away at the age of 49 due to pulmonary heart disease caused by inhalation pneumonia. At that time, there was no distillation technology, and the purity was only over 70% when boiled in a pot. He did not receive any awards. The invention award for arsenic trioxide has been vacant because we are commemorating him - originality is originality, and we must respect science, "Ma Jun added.

03. China CGT: Large scale, low conversion rate

There are over 2000 clinical studies on CGT in tumors worldwide, with China accounting for approximately half. Ten CAR-T products were approved, including one solid tumor. But the problem is that the commercialization of Chinese products has only produced over 6000 cases in five years, while the United States has produced more than 80000 cases - the population of the United States is only a quarter of that of China.

We have always said that cell therapy goes from following, to running in parallel, to leading. I don't admit it. Many of our things are said by ourselves. To truly achieve global advancement, we need to rely on data, evidence-based medicine, and benefit patients

He presented a set of data from the top 100 medical journals worldwide: from 2015 to 2024, the global share of clinical trials for innovative drugs in China increased from 3.2% to 14.9%. China's global share in the CGT field has reached 11%, higher than other tracks. But in the past two years, there has been a "double decline" - not only has the relative share growth in global CGT slowed down, but the relative importance in the domestic biopharmaceutical landscape has also declined.

This is not simply a slowdown in growth rate, but a signal of structural kinetic energy loss.

What worries Ma Jun even more is the stage distribution. In CGT clinical trials in China, phase I accounts for 14.8%, while globally it is only 3.1%; But in Phase II/III, China only has 1.6%, while globally it is 13%. Phase I is five times more than Phase II, and eight times less than Phase II/III. Unstable investment is forcing innovative drugs to fail

The funding sources are also unbalanced, with industry sponsorship only accounting for 25.6% in China's CGT trials, while the global average is 47.3%. A large amount of research relies on public funding support, making it difficult to complete long-term clinical validation.

04. IIT is both an engine and a pain point

There is a significant characteristic of CGT in China - there are particularly many IITs. In the field of CGT, clinical studies initiated by researchers account for 94% of high impact papers. Ma Jun said that this is "a bit upside down" - "It may be more important to make it a medicine and benefit the people, not just for publishing articles

He listed several real challenges: AAV gene therapy for infantile onset Pompe disease and genetic deafness gene therapy, both of which are influential IIT achievements, but none have entered confirmatory key trials or obtained regulatory approval. A large number of high-quality clinical studies are stuck in the early stages.

Regulatory vacuum. IIT has long been limited to pure scientific exploration, with unclear transformation policies and a lack of data standard quality. Everyone in the government departments has the the final say, but nobody has the say. ”
There is another more practical issue, which is unclear legal responsibility. Who is the biggest beneficiary of the dual track system? Who is the biggest risk? Is it the state, the company, or the government that provides the bottom line? There is no definition yet

He said sometimes he feels like he's "shaking the light" - doing both clinical registration and IIT, with different logic, requirements, and endpoints for the two types of research, and unclear paths for how to connect and translate them.

05、Order No. 818, the highly anticipated 'catfish'

In September 2025, the State Council passed Order No. 818, which came into effect on May 1st of this year. Its full name is the "Regulations on the Administration of Clinical Research and Clinical Translational Applications of Biomedical New Technologies".

Regarding the changes brought about by Order 818, Ma Jun summarized them into three points.

Firstly, clinical research has shifted from a defense approval system to a filing system. It has been proven safe and effective through non clinical research, and after academic and ethical review, it can be carried out by filing with the National Health Commission within five working days. There is another solution to the previous deadlock where animal models could not simulate human bodies and could not obtain registration permits.

Secondly, it has been clarified that it can be converted into clinical applications. After approval, it can be charged and implemented. This opens up an opportunity for the industrialization of IIT research results.

Thirdly, regulations have been made on the qualifications and responsibilities of all parties involved. The initiating institution must be a domestic legal entity, the implementing institution must be a tertiary hospital, and the PI must hold a senior professional title. At the same time, it has been clarified that the initiating institution shall bear the treatment costs for health damage, and insurance purchase is encouraged with data retention for 30 years.

The industry compares Order 818 to a "catfish" and expects it to break the conversion deadlock. Ma Jun agrees with this, but he also provides five action suggestions: providing systematic training to the ethics review committee to enhance CGT review capabilities; Maintain regulatory flexibility while ensuring safety; Promote the establishment of a collaborative mechanism and data interoperability platform between the National Health Commission and the National Medical Products Administration; Mandatory public reporting and feedback of clinical research results; Mandatory long-term follow-up registration, patient-centered.

Accurate execution is necessary to unleash the maximum potential of the catfish effect, "he said.

06. Cells or genes? The boundary is being redrawn

Ma Jun also spent a lot of space discussing an issue that is easily overlooked by the industry - the classification method of advanced therapeutic drugs, which will be more clear in the future.

China's latest classification of advanced therapeutic drugs only retains two major categories: cell therapy drugs and gene therapy drugs. Is the core boundary of the division "external" or "internal". Extracorporeal manipulation and transfusion after extracorporeal modification are classified as cell therapy; And drugs administered internally, such as CAR-T, AAV, oncolytic viruses, and mRNA drugs delivered through viral vectors, are all classified as gene therapy.

Traditional individual therapy is called cell therapy, while in vivo therapy is called gene therapy. This is a major update that is clearer. However, it should be noted that regulatory classification mainly serves the drug declaration path, facilitating differentiated guidance for research requirements, but it is not equivalent to a one size fits all technical standard. Specific products also need to comply with technical standards that are suitable for their mechanism of action and risk characteristics, "said Ma Jun.

The boundary between technology and drugs is not simply determined by cell therapy or gene therapy. There are many differences between the two in terms of application path, evaluation criteria and time limit, quality control requirements, and requirements for clinical data. The scope of research and transformation applications is also significantly different. For enterprises, the classification of products directly affects their development strategies and market pace. Ma Jun reminds his colleagues to think carefully about this classification issue during the project initiation stage, otherwise "discovering the wrong path halfway through would be very costly

He specifically mentioned "in-vivo CAR-T" and "regular CAR-T": "I've been talking about it for over a decade, every day. But to become a medicine, it's even more difficult. In recent years, I have not seen any CAR-T drugs in the body. Although I have approved one and enrolled three patients, the future is not bright and it is still very complicated. ”

In vivo CAR-T needs to address a series of issues such as targeted delivery, in vivo amplification, persistence, and safety, each of which is far from mature and cannot be blindly pursued just because of the concept fire.

07. Enterprises and doctors stand together

At the end of the report, Ma Jun shared an American case.

A biotechnology company with only six people has solved the problem of KRAS targets not being able to be used as drugs through the "molecular gel exchange method", and the company's valuation has increased from a six person team to 30 billion or even 300 billion US dollars.

KRAS has mutations in many tumors, but the receptor cannot bind to it. They use molecular gel exchange to enter the nucleus of the cell, bind with DNA, and disrupt tumor growth. At this ASCO meeting, all attendees applauded for six minutes, indicating that the clinical trial has entered phase III. ”

Ma Jun called on the companies, investors, and doctors present to "work together to serve China's cell/gene therapy and innovative drugs

He counted that between 1964 and 2002, 18 clinical doctors who conducted basic research at NIH won the Nobel Prize. History has repeatedly proven that fundamental research on simple model organisms is directly related to human physiology and diseases. Doctors are the primary helpers in translational medicine

As an old doctor who has been working for 55 years, the day when I can see innovative Chinese drugs truly serving patients may be my greatest happiness, "he said in the end.