FDA’s 2026 Flexible CMC Information Signals a New Chapter for Cell and Gene Therapies
On the 11th of January 2026, the U.S. Food and Drug Administration (FDA) released information clarifying flexibility across chemistry, manufacturing, and control (CMC) requirements for cell and gene therapies (CGT).
This announcement does not introduce new regulations or revised statutory requirements. Instead, it shares how existing regulatory flexibility is applied within current CMC frameworks.
The FDA’s Center for Biologics Evaluation and research (CBER) noted that this flexibility has, historically, been exercised on a case-by-case basis and is now being communicated more broadly, so that sponsors have a clearer picture of what may be scientifically acceptable. They expressed that “While there is a long history of making concerted efforts to help sponsors meet standards to assure product safety, purity and potency, the application of flexibilities has not always been fully clear to stakeholders.”
The FDA has been clear that the goal is to support innovation without compromising safety or efficacy. By adopting phase-appropriate controls, lifecycle validation, and more pragmatic release criteria for investigational products, regulators are enabling developers to move faster whilst staying accountable. The result is a framework that reflects how CGT development actually works: iterative, data-driven, and closely linked to manufacturing performance.
As such, the FDA clarified the following approaches to CMC oversight during clinical development:
Full commercial cGMP compliance is not expected before later-phase clinical trials, with manufacturing controls applied in a phase-appropriate manner during early development.
Process and analytical methods are reviewed across the product lifecycle, with validation approaches expected to evolve and be refined as product and process understanding increases.
Final product specifications are not required during early investigational stages, and permissive release criteria may be used for products administered in clinical trials, consistent with investigational use.
Minor manufacturing changes may be introduced during development, with the level of comparability data required determined by the nature of the change and the stage of development.
Why this matters beyond the United States
Although the clarification originates from the U.S. FDA, its influence extends globally. Clearer articulation of lifecycle-based regulation provides an international reference point for how advanced therapies can be governed in a scientifically rigorous, yet operationally realistic way. According to the agency, “CBER is proactively communicating about regulatory flexibilities that were previously applied case-by-case to select CGT therapies. By communicating these approaches broadly, we aim to expedite product development across the CGT field.”
However, timely access to cell and gene therapies depends on more than approvals and policy settings. It requires the ability to manufacture locally, manage quality at scale, and deliver treatments reliably across the health system.
For New Zealand, this means that building capability in key areas is essential to turn scientific progress into practical outcomes for patients:
Manufacturing capability determines access, particularly for therapies that are highly individualised, produced in small batches, or constrained by short shelf lives.
Supply chain resilience is critical for time-sensitive or personalised therapies, where reliance on offshore manufacturing can introduce delays, logistical risk, and increased cost.
Workforce capability and quality systems underpin safe delivery, requiring specialised skills, robust process control, and sustained operational experience across the health system.
Building local capability reduces cost pressure, strengthens supply security, and develops the expertise needed to support long-term innovation. For patients across Aotearoa, particularly Māori, Pacific, and regional communities, local capability is what transforms scientific progress into safe, real-world treatments. Therefore, while regulatory clarity lays the roadmap; local capability drives the journey from science to patient care.
Keeping Pace with Global Standards
The FDA’s clarification on CMC flexibility highlights a pragmatic, science-driven approach to cell and gene therapy development, emphasising iterative, data-informed pathways from early research to licensure. For New Zealand, and for BioOra, this provides a clear reference point as the sector grows, reinforcing confidence in how advanced therapies can be safely developed and evaluated. To fully seize this opportunity, New Zealand must keep pace with international regulatory expectations - turning innovation into timely, globally recognised patient care.