At present, six stem cell therapy projects in China are in the critical Phase III clinical trial stage, which indicates that the field of stem cell drugs is experiencing an important turning point.

Phase III clinical trials are the final threshold before drug approval. Successful approval means that the therapy has proven its safety and efficacy in a larger population. The number of pipelines that have entered the later stage of research and development is significantly higher than before, which concentratedly reflects that the achievements of more than ten years of technological accumulation are beginning to enter the harvest period.

Based on the research and development process, if everything goes smoothly, this batch of drugs is expected to be approved for marketing rapidly between 2026 and 2027, thus creating the first significant marketing boom in the domestic stem cell therapy field. This will be an important milestone for this field to move from exploration to mature commercialization.

The core factors driving this upsurge include: the continuous optimization of technology (such as the standardization of cell production and the improvement of quality control), the gradual clarification of regulatory paths (such as the accelerated approval of the RMAT for Advanced therapies in regenerative medicine established by the FDA), and the promotion of huge unmet clinical needs. The launch of the first batch of commercial products will greatly boost industry confidence and pave the way for more pipelines in the future, ushering in a new era of cell therapy.

一、The field of bone and joint diseases

AlloJoin® (Allogeneic adipose-derived Mesenchymal Stem Cell Injection)

Sponsor: Sibiman Biotechnology (CBMG)

Indications: Knee osteoarthritis (Kellgren-Lawrence II-III grade)

R&d progress

Phase III initiation: In April 2023, it was the first stem cell drug in China to enter Phase III clinical trials for knee osteoarthritis.

Current status: Recruitment has not yet begun (as of July 2025), with a planned sample size of approximately 60 cases.

Experimental Design

Multicenter, randomized, double-blind, positive control, with sodium hyaluronate (a traditional joint lubricant) as the control drug.

Primary endpoint

WOMAC score improvement (48 weeks) : Assessing joint pain and functional recovery.

MRI cartilage quantitative analysis (24 weeks) : Detect changes in cartilage volume (secondary endpoint extended to 104 weeks).

Preliminary data: The 96-week follow-up of Phase II shows:

The pain relief rate was 40% higher than that of the control group, and the rate of cartilage wear was 32% slower.

No serious adverse reactions were reported and the safety was good.

2. IxCell hUC-MSC-O (Human Umbilical Cord Mesenchymal Stem Cell Injection)

Sponsor institution: Shanghai Aisar Biotechnology

Indications: Primary knee osteoarthritis (same as K-L grade II-III)

R&d progress

Phase III initiation: August 2023, with a planned enrollment of 368 cases (the largest-scale Phase III trial of knee osteoarthritis stem cells in China).

Registration status: Not yet publicly displayed on the drug clinical trial platform (the enterprise disclosed that it has been initiated).

Mechanism of action

Regulate the joint immune microenvironment through paracrine factors such as TGF-β and IL-10 to promote chondrocyte regeneration.

Innovation points of the experiment

The "four-blind method" design was adopted (blinding of researchers, subjects, statisticians, and sponsors), with normal saline as the control drug.

Expected advantage: The efficacy of a single injection can last for more than 12 months (Phase II data).

二、Crohn's anal fistula

3. Allogeneic adipose-derived mesenchymal stem cell Injection (DK001

Sponsor institution: Jiangsu Dekang Biology

Indications: Inactive/mild active Crohn's disease complex anal fistula

R&d progress

Phase III status: Received tacit approval for clinical trials from CDE in February 2024 (Acceptance number CXSL2400111), currently recruiting, with a planned enrollment of 240 cases (1 case has already been enrolled).

Mechanism of action

Inhibit the Th1/Th17 inflammatory pathway, promote the proliferation of Treg cells, and accelerate fistula healing.

Preclinical evidence

The rat model showed that the therapeutic effects of the high-dose, medium-dose and low-dose groups were positively correlated, and the inflammatory factor TNF-α decreased by more than 50%.

4. Human TH-SC01 Cell Injection

Sponsor institution: Jiangsu Tuohong Kangheng

Indications: Complex anal fistula in Crohn's disease (indications of the same type)

R&d progress

Phase III initiation: In June 2025, ethical approval was obtained from the leading unit, and multiple centers across the country simultaneously enrolled in the group (with a planned 240 cases and 1 case already enrolled).

International recognition: In July 2025, it won the "Best Poster Award" at the AOCC conference, and the data of issues I/II received high praise from global experts.

Core data (Phase I/II) :

The closure rate of anal fistula: 68% vs 32% in the experimental group vs. the control group (p<0.01).

Safety: No serious adverse reactions, and the stability of the cell preparation reaches 144 hours (supporting flexible clinical application).

三、Liver failure and GVHD

5. Mesenchymal stem cells for injection (umbilical cord

Sponsor: Angsai Cell (a subsidiary of Hanlian Pharmaceutical)

Indications: Acute-on-chronic liver failure (ACLF)

R&d progress

Global status: The world's first stem cell drug to enter clinical trials for the ACLF indication (IND approved in 2021).

Phase III: The IIb/ Phase III study will be initiated in March 2025, in collaboration with 20 top institutions including Xijing Hospital.

Mechanism and Potential

The Phase II data shows that the 28-day survival rate has increased by 25% through anti-inflammatory and liver tissue repair to improve liver function.

6. Human primal mesenchymal stem cells (Pumetosai)

Sponsor institution: Tianjin Madison (Technical support from Peking Union Medical College)

Indications: Graft-versus-host disease (GVHD)

R&d progress

Phase III initiation: In July 2023, the target is to enroll 100 patients with GVHD after bone marrow transplantation.

Technical background: Relying on the technology of the Institute of Basic Medical Sciences of Peking Union Medical College, it is positioned as a "shelf-type" stem cell drug (no matching required).

四、Research and Development Trends and Core Breakthroughs

In the field of knee osteoarthritis

Technical route comparison

Fat source (AlloJoin®) : Its advantage lies in large-scale preparation and maturity, with a focus on structural repair.

Umbilical cord source (IxCell) : It has a stronger differentiation ability and a prominent immunomodulatory effect, and is suitable for moderate to severe cartilage injuries.

Clinical need: Traditional treatments (sodium hyaluronate/hormones) only relieve symptoms, and stem cell therapy is expected to achieve functional reversal.

2. Field of anal fistula treatment

Competitive landscape: Tuohong Kangheng (TH-SC01) and Dekang Bio (DK001) are competing on the same track

TH-SC01: Internationally certified, leading in closure rate data, positioned in the high-end market.

DK001: Focusing on immune regulation, with controllable costs, it is suitable for grassroots promotion.

3. Liver failure and GVHD

Angcel cells: The game-changer in the global ACLF treatment blank area. If the Phase III is successful, it will rewrite clinical guidelines.

Pumetosai: It addresses the second-line treatment dilemma for GVHD patients, but the sample size is relatively small (100 cases), and expanded validation is needed.