Executive Summary
The global cell and gene therapy (CGT) market is experiencing unprecedented growth, projected to expand from USD 25.03 billion in 2025 to approximately USD 117.46 billion by 2034, with a CAGR of 18.7%. Amid this expansion, China has emerged as a crucial market and development hub for CGT innovations, offering strategic advantages that can significantly accelerate clinical development timelines and reduce costs.
This white paper examines how international CGT companies can leverage China's rapidly evolving ecosystem to enhance their global development strategies, and how GCP ClinPlus—China's leading CGT clinical research organization—can serve as the ideal partner for navigating this opportunity.
The Global CGT Landscape: Challenges and Opportunities
Cell and gene therapies represent the frontier of medical innovation, offering transformative and potentially curative treatments for previously untreatable conditions. However, these breakthrough therapies face significant development hurdles:
● Extended Development Timelines: The average CGT development cycle spans 10-15 years
● Enormous Development Costs: Typically exceeding $1.5 billion per successful therapy
● Complex Manufacturing and Logistics: Particularly challenging for autologous cell therapies
● Regulatory Uncertainties: Recent leadership changes at FDA's CBER have created additional uncertainties in the US market
● Pricing Pressures: The Inflation Reduction Act and similar policies are creating new pressure on innovative therapies
For CGT developers seeking to optimize their development pathway, a strategic approach to China can provide compelling advantages that address these challenges directly.
China's Rising Prominence in CGT Development
Recent trends demonstrate China's growing significance in the global CGT landscape:
● China now has approximately 50% more CAR-T therapies in development than US companies
● China's CGT market size reached $2.5 billion in 2025, growing at a remarkable CAGR of 276% from 2020
● The National Medical Products Administration (NMPA) approved 10 CGT products through 2024, including 6 CAR-T cell therapies
● In 2024 alone, China's NMPA received 134 CGT clinical trial applications involving 102 distinct products
● Research targets have exceeded 40, covering 90% of hematologic malignancies and 65% of solid tumors
Regulatory Breakthroughs
Recent policy changes have dramatically improved access for foreign CGT developers:
● In 2023, the CDE released the 'Guidelines for Non-clinical Research of Gene Therapy,' which clarified technical standards to release policy benefits, driving a surge in applications in high-value areas such as solid tumors and autoimmune diseases
● In September 2024, China authorized foreign investment in human stem cell and gene therapy technology development and applications in designated free trade zones (Beijing, Shanghai, Guangdong, and Hainan)
● By December 2024, multiple foreign-invested enterprises had successfully registered to conduct CGT development in China's free trade zones
Patient Access & Reimbursement Evolution
A critical barrier to CGT adoption—reimbursement—is being systematically addressed:
● Multiple CAR-T products have been included in China's "HuiminBao" insurance programs across various regions
● Risk-sharing payment models pioneered by companies like FosunKite are establishing viable payment pathways
● China has announced plans for a "Class C" medical insurance category specifically designed for high-value innovative therapies like CGT
● CGT products are increasingly covered by commercial insurance products
Strategic Advantages of China for Global CGT Development
China offers several distinct advantages that can accelerate CGT development, particularly as CGT applications expand beyond hematologic malignancies to solid tumors:
1. Accelerated Patient Recruitment
● Access to treatment-naïve populations: 73% of trials with treatment-naïve cancer patients are conducted in China
● Large patient pools: China's population base provides access to significant numbers of patients with conditions targeted by CGT, including:
○ Acute lymphoblastic leukemia (ALL): Incidence of 0.69/100,000
○ Acute myeloid leukemia (AML): Incidence of 1.62/100,000
○ Lymphoma: 6.66/100,000; 85,000 new cases in 2022, Non-Hodgkin lymphoma (NHL) accounts for 91% of all lymphoma cases in China
○ Multiple myeloma: Incidence of 1/100,000
○ Mortality data shows 42,000 deaths from lymphoma and 50,000 deaths from leukemia annually
2. Significant Cost Advantages
● Clinical trial costs are 60-80% lower than in Western markets
● Unlike Western markets, China's trial costs are not experiencing significant inflation
3. Rapid Study Timelines
● Patient recruitment is often 2-3 times faster than US/EU studies
● Parallel ethics committee and regulatory reviews accelerate study initiation
4. Robust Clinical Infrastructure
● Over 1,000 CGT clinical trials initiated by May 2023
● Well-established CAR-T manufacturing and administration capabilities
● Growing CDMO ecosystem with high-quality manufacturing standards
Addressing Common Concerns for US/EU Companies
We understand that foreign companies may have hesitations about conducting CGT studies in China. These concerns require specialized expertise to navigate:
Logistics and Manufacturing
Foreign companies typically face 7-8 week turnaround times for cross-border cell processing versus just 4 weeks for locally manufactured products. Strategic local partnerships can address this gap.
Regulatory Complexity
Successful navigation requires expertise in:
● Document preparation specific to Chinese requirements
● Human Genetic Resources Administration of China (HGRAC) approvals
● Import/export procedures for biological materials
● Dual-track regulatory pathways for investigator-initiated trials (IITs) and industry-sponsored studies
Intellectual Property Protection
While China has strengthened IP protection, CGT developers need clear strategies to address requirements for technology localization while protecting core IP.
Quality and Data Standards
Ensuring consistent quality standards across global development programs requires partners with deep experience in both Chinese and global regulatory environments.
Successful Global-China Integration Models
Several leading global CGT companies have successfully navigated China's ecosystem:
● Gilead's Yescarta: Co-developed with FosunKite specifically for the China market, leading to NMPA approval
● Legend Biotech: Partnered with Janssen to develop Carvykti, which received both FDA approval and Breakthrough Therapy Designation in China
Strategic Approaches to China-Global Integration
International CGT developers can consider three primary approaches to integrating China into their global development strategy:
1. Multi-Regional Clinical Trial (MRCT) Strategy
The deepest integration approach, where China participates from early development stages:
● Chinese sites included in global trials
● Data satisfies both global and Chinese regulatory requirements
● Enables simultaneous regulatory submissions
2. Extension Study Approach
A hybrid model:
● China joins global pivotal studies
● Some independent Phase 1 work conducted in China
● Leverages global efficacy data while generating China-specific safety data
● Aims for simultaneous submissions with some China-specific components
3. Bridging Study Strategy
A sequential approach:
● Global development proceeds first
● Followed by specific bridging studies in China
● Demonstrates applicability of global data to Chinese patients
● Results in "catch-up" submissions to Chinese regulators
GCP ClinPlus: Your Bridge to China's CGT Ecosystem
As China's leading clinical CRO specializing in cell and gene therapies, GCP ClinPlus offers unique capabilities to help navigate this complex landscape:
Unparalleled CGT Experience
● Conducted 50+ IIT, Phase I-III CGT clinical studies across multiple indications including hematologic malignancies, solid tumors (primary liver cancer, advanced solid tumors), and genetic disorders
● Supported the first stem cell therapy NDA in China
● Supported the first gene therapy for thalassemia in China
● Leadership team has supported 10+ cell, gene, and RNAi NDAs
● Biometrics team has supported four RNAi NDA approvals from FDA and EMA
Comprehensive CGT Services
Our Gene and Cell Therapy Division provides end-to-end support:
1. Strategic partnership planning to optimize China CGT development approaches
2. Full-service clinical trial management with accelerated patient recruitment
3. Integrated biometrics services supporting global regulatory submissions
4. Regulatory strategy consultation for optimal China and global development pathways
5. Site selection and investigator engagement through our established network
Distinctive Capabilities
● Deep connections with leading hospitals and experienced investigators
● Streamlined processes to minimize administrative burdens
● Local production partnerships to avoid cross-border cell processing delays
● Expertise in site selection focusing on first-tier hospitals with established CGT experience
● Global biometrics support from over 200 biostatistics professionals
Conclusion
As the CGT landscape continues to evolve, integrating China into global development strategies offers compelling advantages in terms of cost, speed, and access to patients. With its unparalleled experience in CGT clinical development, GCP ClinPlus is uniquely positioned to help international biotechnology companies navigate the complexities of China's ecosystem while maintaining the highest global quality standards.
By partnering with GCP ClinPlus, global CGT developers can accelerate their development timelines, optimize costs, and potentially access the world's second-largest pharmaceutical market—ultimately bringing transformative therapies to patients more quickly and efficiently.
(Data cited in this article is sourced from Precedence Research, Frost & Sullivan research, the China National Medical Products Administration (NMPA) website, and the Chinese Journal of Oncology.)