CTGT/ATMP clinical trials surge in China, as first stem cell therapy product is conditionally authorized

On 7 September 2024, China's NMPA, jointly with other authorities, issued the “Notice on Carrying Out Pilot Programs to Expand the Opening-up in the Healthcare Sector,” which permits foreign-invested companies to engage in human stem cell, gene diagnostics, and therapeutic technology development and application within free trade zones in Beijing, Shanghai, Guangdong, and Hainan for product registration and manufacturing. Since its issuance, local governments in these cities or provinces have been progressively rolling out supportive policies, facilitating the market entry of products derived from such cutting-edge technologies.

Complementing these openness initiatives, the NMPA is also working to strengthen the foundational governance of CTGT products by clearly defining key terms and the scopes of these therapies. On 10 June 2025, the CDE released the “Draft Guidelines on the Scope, Classification, and Definition of Advanced Therapy Medicinal Products (ATMPs)” for public consultation. By proposing to classify eligible cell therapies, gene therapies, and emerging modalities such as tissue-engineered products under the internationally recognized term “ATMPs”, the draft aims to clarify regulatory boundaries, enable risk-proportionate oversight, and align with global practices (albeit with certain localized distinctions). Once finalized, this framework will provide much-needed clarity for developers and pave the way for more efficient review and lifecycle management of innovative therapies. To further accelerate the development of high-potential therapies, on 8 September 2025, the NMPA issued the “Guidelines for Class I Meeting Applications and Management in Communications on ATMPs” also for public consultation, which aim to facilitate timely regulatory feedback for sponsors, helping to reduce development risks and shortening timelines. Following the finalization of these documents, we expect a series of upcoming technical guidelines, risk-based supervision policies, and lifecycle management frameworks dedicated to ATMPs in the near future.

Despite these progressive reforms, foreign companies continue to face significant challenges in China. Chief among them is the evolving and increasingly complex legal landscape, particularly in areas such as scientific ethics, data governance, and lifecycle compliance. Additionally, competitive pressure from domestic pharmaceutical companies, often bolstered by government incentives and cost advantages, can be formidable. Compounding these issues are geopolitical tensions, especially with major economies like the U.S. and the EU, which may complicate technology transfer, cross-border collaboration, supply chain, and import/export strategies. In this context, close monitoring of global policy shifts and strategic agility will be essential for foreign stakeholders seeking sustainable growth in China's dynamic CTGT/ATMP sector.

Authored by Roy Zou, Lu Zhou, Jessie Xie, and Olivia Xie