De-risking Cell and Gene Therapy Development: Global Regulatory Strategies for Streamlined Development
Dr. Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research (CBER), recently shared insights at the 2025 JP Morgan Healthcare Conference, outlining the FDA’s ongoing efforts to accelerate advances in cell and gene therapy (CGT). With significant breakthroughs in 2024—including approvals for tissue-engineered products, mesenchymal stem cell therapies, and solid tumor-targeting T-cells—the CGT sector continues to make significant progress. However, despite these advances, challenges in regulatory harmonization, market access, and cost-effectiveness persist, requiring strategic policy measures and industry collaboration to de-risk CGT development in the US and beyond.
The Current CGT Landscape in the US
The US remains a leader in CGT innovation, with the FDA approving 24 cell and gene therapies to date, and is predicted to review or receive applications for a record 15 new therapies in 2025.
Figure 1. Showing FDA-approved and pipeline cell and gene therapies.
Recognising the need for new approaches to regulating these innovative therapies, Dr. Peter Marks discussed several new initiatives underway at the FDA. Key initiatives include:
Risk-based regulatory frameworks: The FDA is exploring a new approach to human cells, tissues, and cellular-based products (HCTPs), addressing over- and under-regulation issues with a planned scientific workshop in February 2025.
Platform technologies like CRISPR: Marks emphasized the need for a regulatory shift, treating CRISPR as a platform tool rather than a product, which could streamline development and approval processes, potentially under the draft platform technology guidance.
Rare disease initiatives: Programs such as the Rare Disease Hub and collaborative efforts with the EMA are expected to accelerate treatment pathways for underserved conditions.
While these regulatory-focused initiatives are promising, ongoing efforts should also focus on reducing manufacturing bottlenecks, ensuring sustained funding for innovation, and fostering public-private partnerships to sustain momentum in the sector.
The UK Approach: A Strategic Roadmap for Growth
The UK’s CGT ecosystem is supported by institutions such as the Cell and Gene Therapy Catapult and the Advanced Therapy Treatment Centres (ATTC), which focus on scaling up manufacturing and integrating therapies into the National Health Service (NHS). Recent milestones include:
NHS adoption of CRISPR-based therapies: The approval of Casgevy for transfusion-dependent beta-thalassemia demonstrates the UK’s leadership in pioneering CGT adoption within public healthcare.
Regulatory flexibility: The Medicines and Healthcare products Regulatory Agency (MHRA) is actively harmonizing with global frameworks to enable smoother market entry.
By aligning with the UK’s emphasis on infrastructure and workforce development, other markets can strengthen their own pathways to commercialization.
Germany’s National Strategy: Balancing Innovation with Compliance
Germany is positioning itself as a European leader in CGT with the introduction of a National Strategy for Gene and Cell Therapies, launched in June 2024. This strategy focuses on:
Regulatory streamlining: Efforts to simplify legal pathways and align with the European Medicines Agency (EMA).
Investment in infrastructure: Enhancements to Germany’s biomanufacturing capabilities to ensure scalable production of CGT products.
Industry collaboration: Strong ties between government bodies and industry stakeholders to foster a competitive environment.
Other countries can learn from Germany’s comprehensive approach by expanding infrastructure investment and creating strategic alliances to enhance manufacturing scalability.
Japan’s Regulatory Flexibility: A Model for Rapid Market Access
Japan’s regulatory framework for regenerative medicine, governed by the Pharmaceuticals and Medical Devices Agency (PMDA), may represent one of the most progressive models globally. With a conditional and time-limited approval pathway, Japan has enabled rapid commercialization of therapies such as CAR-T cell treatments. Key elements of Japan's success include:
Accelerated approval pathways: Conditional approvals based on preliminary efficacy data, allowing early patient access.
Post-market surveillance: Rigorous follow-up requirements ensure long-term safety and efficacy.
Public-private collaboration: Strategic partnerships between government, academia, and industry have accelerated innovation.
To remain competitive, regulatory bodies in other regions could consider adopting Japan’s adaptive regulatory approach for certain high-priority therapies, thereby accelerating patient access without compromising safety.
Opportunities for Global Regulatory Synergy
Despite regional differences, there are clear opportunities to align regulatory processes across key markets:
Harmonized Regulatory Frameworks: The FDA, EMA, MHRA, and PMDA could collaborate on common standards for CGT approvals, reducing redundancies and facilitating global market entry.
Data-Sharing Initiatives: Establishing global databases, leveraging federated learning models to share real-world evidence can enhance regulatory decision-making and improve patient safety outcomes.
Streamlined Manufacturing Guidelines: Aligning quality and manufacturing standards globally can reduce costs and accelerate production scalability.
Stakeholders across the CGT ecosystem should work toward strengthening partnerships with international regulatory bodies to create a more unified approach to CGT approvals and commercialization.
A Call for Strategic Action
As CGT continues to revolutionize medicine, proactive steps are needed to de-risk development by:
Modernizing regulatory frameworks to accommodate platform technologies.
Increasing funding for infrastructure and workforce development.
Strengthening international regulatory cooperation to facilitate global market access.
Addressing cost barriers to ensure broad patient access.
By drawing from strategies across the US, the UK, Germany, and Japan, the global CGT sector can create a more resilient and competitive landscape, ensuring that transformative therapies reach patients faster and more efficiently.
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