FDA’s 2026 Guidance Signals a New Chapter for Cell and Gene Therapies
On the 11th of January 2026, the U.S. Food and Drug Administration (FDA) released new guidance that materially changes how cell and gene therapies (CGT), including autologous CAR‑T products, are regulated. This guidance introduces greater flexibility across chemistry, manufacturing, and control (CMC) requirements allowing standards and data expectations to evolve as programmes progress through development. It formalises approaches such as broader early‑stage release criteria, lifecycle‑based validation, and adaptive specifications, recognising that therapies made for individual patients cannot be regulated in the same way as mass‑produced pharmaceuticals.
This is not a lowering of standards; it is a modernisation of them. 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.
The impact of this guidance extends well beyond the United States. By formalising flexibility that was previously agreed on a case-by-case basis, the FDA has set a clear benchmark for modern regulation of advanced therapies. Other regulators will need to watch closely. For international developers, particularly those working with automated or decentralised manufacturing models, this provides clearer expectations and earlier regulatory engagement. For New Zealand, it sends a clear signal that standing still is not an option.
New Zealand has already signalled the need for change, most notably through the Medicines Amendment Bill 2025, which aims to streamline access to overseas-approved medicines and improve regulatory efficiency. However, access on paper does not guarantee access in practice. Without domestic manufacturing capability and regulatory frameworks that support controlled change, advanced therapies remain costly, logistically complex, and out-of-reach for many potential patients. The FDA’s approach highlights the next step: combining regulatory confidence with operational flexibility.
Equitable 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. 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, this capability is what turns scientific progress into real-world outcomes.
For BioOra, the FDA’s guidance reinforces the direction we are already taking in collaboration with the Malaghan Institute of Medical Research. Our focus on automated, scalable CAR-T manufacturing, built around robust quality systems from the outset, aligns closely with the lifecycle-based approach now being endorsed internationally. It affirms that New Zealand-based programmes can be designed to meet global expectations by intent, not as an afterthought.
The FDA’s announcement reflects a broader shift in how advanced therapies are understood and governed. Innovation and safety are no longer treated as competing priorities, but as outcomes that must progress together. For New Zealand, the opportunity is clear: align regulation, infrastructure, and ambition so that therapies discovered here can also be developed, manufactured, and delivered here. The science has moved. The world is adjusting. We need to move with it.