Advanced Therapies in 2026: Navigating New Gene and Cell Therapy Regulations

Cell and gene therapies continue to transform the treatment landscape, and by 2026, both

the FDA and the European Union will have updated frameworks that reshape how these

advanced therapies are developed, approved, and monitored. Companies working in this

space must prepare for evolving expectations around safety, manufacturing, and long term

follow up.

In Europe, the upcoming pharmaceutical legislation reform includes several updates to the

Advanced Therapy Medicinal Products (ATMP) framework. Among these are changes to the

hospital exemption for ATMPs, refined guidance on manufacturing standards, and new

pathways to encourage the development of innovative gene therapies. Updates to the

orphan drug designation rules are also expected, focusing on targeting true unmet medical

needs rather than broad disease categories.

In the United States, the FDA is expanding its guidance on cell and gene therapy oversight,

reflecting lessons learned from the increasing number of approved products in oncology,

rare diseases, and genetic disorders. New guidance documents emphasize long term safety

monitoring, often requiring 15 year follow up studies for gene therapy recipients. The

agency is also focused on Chemistry, Manufacturing, and Controls (CMC) expectations,

ensuring that complex manufacturing processes are consistent and reproducible, minimizing variability and ensuring product quality. Additionally, the FDA is strengthening recommendations for post-market surveillance, risk mitigation strategies, and patient registries to track both efficacy and adverse events over time. This comprehensive approach aims to balance rapid access to innovative therapies with the highest standards of safety and reliability.

For developers, the implications are significant. Regulatory bodies will expect stronger

evidence of durability, better control of manufacturing variability, and clearer

communication of risk benefit data. Companies will also need to plan earlier for post market

commitments, including patient registries and long term safety assessments.

With these changes, 2026 is shaping up to be a defining year for the regulation of advanced

therapies. Organizations that invest now in scalable manufacturing systems, robust data

management, and proactive regulatory engagement will have a clear advantage when the

new frameworks are fully implemented.

If your company is developing gene or cell therapies and needs expert guidance on meeting

the evolving regulatory requirements, PRP Compliance can help you navigate FDA and EU

expectations and position your program for long term success.