Core Insights - The article discusses the rapid development of cell and gene therapy (CGT) in China, highlighting its transition from a follower to a parallel and even leading position in the global market [1][2]. Industry Overview - The global CGT clinical trials have reached approximately 2000, with China accounting for over 50% of these trials and over 60% in the cell therapy sector [1]. - China has approved 14 CGT products, including 7 CAR-T products, 3 small nucleic acid products, 2 gene therapies, 1 stem cell therapy, and 1 oncolytic virus therapy, with 9 of these approved in the last three years [2]. Policy and Regulatory Environment - Recent policies, such as the CDE's draft on advanced therapy products, aim to clarify definitions and accelerate innovation, attracting investment and enhancing international competitiveness [2][4]. - The National Medical Products Administration (NMPA) has introduced a 30-day review process for innovative drug clinical trial applications, improving the efficiency of drug approvals [3]. Clinical Development and Innovation - The CGT sector is experiencing a surge in clinical trial approvals, with 765 new drug clinical trial applications reviewed by the CDE as of mid-2025 [5]. - The introduction of AI-driven platforms, such as the NanoForge by Jitai Technology, represents a significant technological advancement in CGT delivery systems [5]. Market Dynamics and Challenges - CGT products are currently expensive, with CAR-T therapies costing over one million yuan per treatment, raising concerns about accessibility for patients [6][7]. - The market is dominated by multinational companies, but Chinese firms are rapidly emerging through differentiated innovation and international collaboration [6]. Future Prospects - The potential expansion of cell therapies into solid tumors could significantly increase the patient population benefiting from these treatments, although challenges remain in addressing the complexities of tumor microenvironments [8]. - Gene therapy may see a reduction in costs and an expansion of market opportunities as it moves towards treating more common diseases, but it faces intense competition in these areas [9].

Core Viewpoint - The article discusses the establishment of a regulatory framework for advanced therapy medicinal products (ATMPs) in China, aiming to enhance the quality of the industry and align with international standards [1][2][4]. Group 1: Definition and Classification - The draft document defines "advanced therapy medicinal products" as those developed, produced, and regulated according to pharmaceutical regulations, including cell and gene therapies [2][4]. - ATMPs in China are categorized into three main types: cell therapy medicinal products (CTMPs), gene therapy medicinal products (GTMPs), and others based on their material basis and active ingredients [2][6]. Group 2: Industry Development and Global Position - The advanced therapy sector is identified as a core area of life sciences, crucial for fostering future industries and promoting high-quality development in the bio-economy [3][4]. - As of 2024, China has made significant progress in CGT drug development, with 4 out of 17 global CGT therapies approved, and 6 out of over ten CAR-T products approved worldwide [3][4]. Group 3: Regulatory Challenges and International Coordination - The current lack of regulatory definitions and industry consensus in China poses challenges for the development, technical review, and regulation of advanced therapies [4][6]. - The draft aims to unify the naming, scope, and classification of advanced therapy products, facilitating faster reviews and approvals while enhancing international regulatory coordination [1][4]. Group 4: Specific Regulatory Provisions - The draft specifies that certain products, such as blood components and reproductive cells, are excluded from the definition of ATMPs [6]. - CAR-T therapies are classified as "ex vivo gene-modified cell products," differing from classifications by other global regulatory bodies, reflecting a focus on the material basis and characteristics of the products [6][7]. Group 5: Future Prospects and Inclusivity - The classification framework is designed to be forward-looking and inclusive, allowing for the integration of new technologies and products as they develop [7]. - The draft has incorporated feedback from over 70 industry experts and representatives, indicating a collaborative approach to regulatory development [7].