Cell Notes:  What FDA’s Regulatory Flexibility Means for the Future of Cell and Gene Therapy

In her monthly column "Cell Notes," AABB's Christina Celluzzi, PhD, MS, CABP(H), shares insights, findings and commentary on emerging topics in biotherapies. This piece launches a three-part “Cell Notes” series examining how regulatory flexibility is reshaping cell and gene therapy (CGT) development, delivery and the workforce that supports it. Subscribe to CellSource to receive "Cell Notes" and the latest biotherapies updates from AABB directly in your inbox. 

“Regulatory flexibility” is appearing more often in discussions across CGT as the field continues to expand and mature.

Flexibility does not mean reduced standards. The U.S. Food and Drug Administration has emphasized that expectations for safety, quality and patient protection remain unchanged. Rather, regulatory flexibility reflects recognition that CGT development is inherently iterative. Manufacturing processes evolve, analytical methods mature and clinical experience informs refinement over time. Regulatory pathways are adapting to reflect that reality.

The pace of activity through 2025 helps explain this shift. FDA continues to oversee a growing and increasingly complex pipeline, with multiple novel cell and gene therapies approved. Additionally, we see the continued and expanded use of expedited programs such as regenerative medicine advanced therapy (RMAT) designation. RMAT is intended to support therapies for serious or life-threatening conditions by enabling earlier and more frequent interaction with FDA while maintaining statutory standards for safety and effectiveness. Together, these trends point to a regulatory framework designed to support innovation while maintaining rigorous oversight.

For developers, this approach can support earlier clinical entry without requiring fully optimized manufacturing at the outset, allowing learning from early clinical experience to inform how therapies are ultimately delivered to patients. Phase-appropriate expectations allow process improvements and analytical refinement to be incorporated over time. In practice, early processes do not need to be perfect, but they do need to be understood, controlled and clearly justified. Clear documentation of rationale, risk and planned evolution becomes central to development strategy.

Clinical teams and patients may benefit as well. More adaptable pathways can enable earlier trial initiation and, in some cases, faster access to innovative therapies, particularly for rare or serious health conditions.

Regulatory flexibility still requires discipline. Early engagement with FDA, coordination across clinical, manufacturing, quality and regulatory teams, and thoughtful change management remain essential. Rather than simply accelerating timelines, flexibility favors preparedness and places growing importance on a skilled and adaptable workforce—one capable of learning alongside the science and advancing therapies responsibly from concept to care.

A closer look at what this shift requires of CGT teams in practice will be explored in an upcoming “Cell Notes” column.

Additional information on FDA’s approach to regulatory flexibility in CGT, including RMAT designation and phase-appropriate manufacturing development, is available from FDA.