China’s CGT Surge: Unpacking A Nature Review on Regulatory Reform, Pipeline Trends & Global Implications

Based on the July 31, 2025 article in Nature Reviews Drug Discovery (Liu et al., CDE & Tsinghua University)

🚀 Overview

China is emerging as a global player in cell and gene therapy (CGT), with a growing number of investigational new drug (IND) submissions, accelerated approval pathways, and a maturing clinical pipeline. A recent review by the Center for Drug Evaluation (CDE) and Tsinghua University, published in Nature Reviews Drug Discovery, outlines the development trajectory from 2017 to mid-2025.

This summary from SinoDrugWatch unpacks the article’s findings and adds critical perspectives on regulatory risks, data quality, and global relevance.

🏛️ 1. Regulatory Reform Fuels Acceleration

China’s CGT regulatory system is anchored by the 2019 Drug Regulatory Science Action Plan, which introduced technical guidelines covering chemistry, manufacturing and controls (CMC), nonclinical studies, clinical trials, and post-marketing surveillance.

Recent milestones include:

• The “One Company, One Policy” model (2024–2025): tailored regulatory pathways for CGT innovators
• 30-day IND fast-track pilot for CGT and rare disease therapies
• Faster review timelines (down from ~260 days to ~60 days for eligible products)

These policies have significantly improved early development efficiency and positioned China as a faster-launch market for advanced therapies.

Critical Perspective:
While fast-track approvals de-risk early timelines, they also shift responsibility for safety validation to post-marketing. Many conditional approvals are based on single-arm trials with short follow-up, raising concerns about durability, particularly for global regulators.

📈 2. IND and NDA Landscape (as of June 2025)

• Total CGT INDs submitted: 765
• INDs approved: 553
• INDs rejected: 214

Rejections largely due to weak scientific rationale, insufficient data, or ethical concerns.

Breakdown by modality:

• CAR-T therapies: 72 exploratory trials, 15 confirmatory trials, 7 NDAs submitted, 9 NDAs approved
• Stem cell therapies: 44 INDs approved, 87 exploratory trials, 3 confirmatory trials, 1 product approved (GVHD)
• Gene therapies: 55 exploratory trials, 7 confirmatory trials, 2 NDAs submitted, 1 product approved (hemophilia B)

Notable Products:

• Axicabtagene ciloleucel (axi-cel), China’s first approved CGT product (June 2021), is a CAR-T therapy for lymphoma. Its approval was based on a bridging study supported by robust global clinical data, setting a precedent for integrating international evidence with local trials.
• Dalnacogene ponparvovec, a gene therapy for hemophilia B, obtained full NMPA approval—a landmark in expanding CGT beyond oncology into rare genetic diseases.

Critical Perspective:
The high rejection rate (28%) highlights maturing regulatory expectations but also ongoing weaknesses in preclinical data packages. Trial designs vary widely in quality, with many still relying on investigator-initiated trials (IITs) that may not meet global data standards.

✅ 3. Approved Therapies and Market Access

By mid-2025, China has approved nine CGT products, including:

• 6 CAR-T products (9 total indications; 7 conditional, 2 full approvals)
• 1 stem cell product (GVHD, conditional approval)
• 1 gene therapy (hemophilia B, full approval)

Key Highlights:

• Most CAR-T approvals are based on single-arm trials with tumor response endpoints.
• Approvals require confirmatory trials within four years, lifelong patient follow-up, and real-world data collection.
• Post-marketing monitoring includes surveillance for secondary malignancies, though no cases have been reported domestically.

Critical Perspective:
The conditional approval pathway is pragmatic but may not satisfy FDA or EMA without further data. Bridging studies and randomized trials may still be required for international acceptance. Surveillance systems for long-term risks like secondary cancers are still developing.

🌱 4. Emerging Indications and Technologies

China’s CGT innovation is expanding beyond hematologic malignancies into:

• Autoimmune diseases: CAR-T therapy has shown remission in systemic lupus erythematosus (SLE) patients.
• New targets: CLDN18.2 (gastric cancer), CD276 (solid tumors), BCMA (multiple myeloma).
• Gene therapy programs: Focused on rare diseases, inherited retinal disorders, and metabolic conditions.

Critical Perspective:
While exploratory programs in autoimmune and rare diseases show scientific potential, they are largely in early-stage development. Long-term efficacy, patient selection, and scalable manufacturing remain open questions.

🌍 5. Global Readiness and Data Quality

China’s CGT industry is growing in clinical volume, but global expansion requires rigorous evidence.

• Over 60% of trials are IITs — often lacking in GCP compliance, robust endpoints, or standardization.
• Multi-regional clinical trials (MRCTs) remain limited.
• Human Genetic Resource (HGR) compliance continues to affect global sample sharing and development.

Critical Perspective:
Many CGT datasets may be insufficient for global regulatory submission without additional pivotal trials. Domestic approval does not guarantee FDA or EMA acceptance, and out-licensing opportunities may depend on bridging data quality and CMC harmonization.

🧭 Summary: Strengths, Gaps & What’s Next

Outlook:
China’s CGT sector is positioned for continued growth, but data quality, long-term efficacy, and regulatory harmonization will be key to realizing global potential. As more programs enter confirmatory phases (2026–2028), global partnerships will increasingly hinge on trial design and real-world outcomes.

📚 References

·       Liu Y, et al. Trends in the development of cellular and gene therapy in China. Nature Reviews Drug Discovery. July 31, 2025.

·       National Medical Products Administration (NMPA). Drug Regulatory Science Action Plan (2019).

·       State Council. Document No. 53: Deepening Drug & Device Reform (2024)

·       Chen Z, et al. Chinese innovative drug R&D trends in 2024. Nature Reviews Drug Discovery. 2024.