Janet MacPherson, PhD
Senior Editor, ISCT Telegraft
NSW Health Pathology
Australia
In 2025, the regulatory landscape for cell and gene therapies (CGT) has taken a turn that reflects the sector’s rapid maturation. Regulators are facing challenges to keep pace with the need to balance innovation with safety, scalability, and equitable access in a timely manner. Changing Laws can be a slow process.
The U.S. Food and Drug Administration (FDA) has taken a proactive stance by releasing three CGT Draft Guidance for Industry documents in September 2025, aimed at allowing innovation while maintaining rigorous standards for patient safety.
These include:
Last year the FDA launched the Gene Therapies Global Pilot Program - Collaboration on Gene Therapies Global Pilot (CoGenT), a pilot initiative designed to explore concurrent, collaborative regulatory reviews of gene therapy applications with international regulatory partners like the European Medicines Agency (EMA). The program aims to increase regulatory harmonization, improve review efficiency, reduce delays, and ultimately accelerate the global access of gene therapies to patients. Modelled after the FDA's Project Orbis for oncology, CoGenT allows foreign regulators to participate in FDA review meetings and share information, starting with initial submissions and potentially expanding to earlier development stages. This initiative is expected to reduce duplication, accelerate approvals, and facilitate global access to life-saving therapies. Time will tell how effective this approach has been.
Regulators are also embracing Artificial Intelligence (AI) and data analytics to manage the complexity of CGT manufacturing and patient monitoring. As therapies become more personalized, oversight must evolve to track individual outcomes, manufacturing variability, and long-term effects. This is following a trend our ISCT Industry Committee has been exploring on how AI can enhance the field. The Committee is hosting a webinar on 15th October on “Introduction of AI/ML in Cell & Gene Therapy: Definitions and Data Standardization”. This is just the latest in a series of ISCT educational sessions on AI-related topics and if you missed it there is a summary from ISCT 2023 in Telegraft.
Companies like Janssen have developed augmented intelligence systems that use machine learning to scan global databases for relevant regulations. These systems can process up to 9,000 regulations per day with over 85% accuracy, dramatically reducing the time required for manual review.
Tools like ChatGPT and other generative models are being used to draft clinical trial protocols, investigator brochures, regulatory submissions and training modules. These models can reference prior documents, reuse content, and generate new text, cutting cycle times and improving consistency.
Natural Language Processing (NLP) tools are now used to analyze FDA and EMA inspection reports, warning letters, and scientific literature. This helps sponsors identify compliance trends and anticipate regulatory risks
In January 2025, the FDA released draft guidance ‘Considerations for the Use of Artificial Intelligence To Support Regulatory Decision-Making for Drug and Biological Products’ on using AI to support regulatory decision-making. It outlines a risk-based credibility assessment framework to ensure AI models used in drug development are trustworthy and fit for purpose.
Hurdles remain:
- Data privacy and security: Sensitive patient and trial data must be protected.
- Model transparency: Regulators demand explainable AI, especially in safety-critical decisions.
- Human oversight: AI outputs must be validated by experts to ensure accuracy and ethical compliance
As cell therapy continues to reshape medicine, AI is poised to reshape regulation. By automating interpretation, enhancing compliance, and enabling smarter decision-making, AI is not just a tool it has the potential to become a strategic enabler of innovation. The challenge is to ensure that AI is used responsibly, transparently, and collaboratively, so that regulatory systems evolve in step with the therapies they govern.
Despite regulatory progress, significant hurdles remain. The high cost of CGT products poses ethical and logistical challenges. Regulators are increasingly involved in shaping value-based reimbursement models and encouraging public-private partnerships to expand access. This works in some regions like the US but is generally not so effective in resource poor or emerging economies. In these situations, the cost to manufacture and deliver CGT therapies needs to be minimised up front rather than trying to reduce the cost to deliver an expensive product.
Manufacturing and distribution infrastructure for CGT products remains fragmented. Regulatory bodies are pushing for standardized protocols and for centralized manufacturing hubs to ensure consistency and scalability. However, there is also a recognition that costs and turnaround times can be improved through adoption of decentralised models (UK). However, despite general agreement on the benefits of local solutions, Regulatory agencies are constrained by the regulations enshrined in law.
2025 marks a pivotal year in the regulation of cell therapies. Agencies are embracing flexibility, global collaboration, and technology-driven oversight. Yet, to fully realize the potential of these therapies, regulators must continue addressing manufacturing, access, and harmonization challenges. The future of cell therapy depends not only on scientific innovation but on the ability of regulatory systems to evolve, ensuring that transformative treatments reach patients safely, swiftly, and equitably. Current regulation should not be a barrier to innovation or patient access. Industry and Product developers have a key role to play. Industry provides a catalyst for therapeutic innovation. As the field continues to evolve, the challenge will be to ensure that these transformative therapies reach all who need them—safely, swiftly, and sustainably.
references
- FDA Guidance on Innovative Trial Designs – FDA.gov
- EMA Clinical Trials Regulation – EMA.europa.eu
- FDA Postapproval Monitoring Guidance – FDA.gov
- Harmonization Challenges – AJMC
- Manufacturing Models – BioSpace
- Regulatory Bottlenecks – HMPI
- Global Harmonization Roadmap – ICH
- FDA Guidance on AI for Regulatory Decision-Making – FDA.gov
- Janssen’s Augmented Intelligence System for Regulation Mining – Janssen.com
- Generative AI in Regulatory Writing – Certara.com
- AI in CDMO Selection – OutsourcedPharma.com
- Indegene on Generative AI in Regulatory Affairs – Indegene.com
- UBC on AI in Pharmacovigilance – UBC.com
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