Key Takeaways:
- Manufacturing processes of cell and gene therapies (CGTs) may remain fluid well into development and even after FDA approval, and therefore sponsors of CGTs may benefit from patent strategies that account for innovations that emerge later and carry independent value from an earlier process.
- Greater regulatory acceptance of iterative manufacturing changes by the FDA may increase the value of innovation beyond final commercial configurations.
- In IP diligence and landscape analyses, practitioners should be mindful that regulatory flexibility may permit multiple manufacturing pathways to support a single approved product, each with potentially distinct IP considerations.
On January 11, 2026, the U.S. Food and Drug Administration announced a clarification to how it applies flexibility to the requirements on the chemistry, manufacturing, and controls (CMC) for cell and gene therapies (CGTs).[1] According to the FDA, this flexibility, which is already partially in practice, is intended to better align regulatory expectations with the medical realities of individualized, and often small-batch, biologics.[2]
The announcement comes at a moment when public attention is increasingly focused on personalized therapies. Earlier this year, widespread media coverage followed the story of an Australian entrepreneur who helped design a customized mRNA cancer vaccine for his dog using tumor sequencing and artificial‑intelligence tools.[3] While the story sparked debate about feasibility, scalability, and regulation, it also underscored that highly individualized therapies are no longer theoretical or academic. Further, the inherent complexity of CGTs presents unique challenges during clinical development that traditional regulations were not designed to accommodate.
The FDA’s January 2026 announcement signals how the agency intends to evaluate CMC issues across the product lifecycle for CGTs. The FDA organized its communication into three buckets: (1) clinical development flexibilities; (2) commercial specification flexibilities; and (3) process validation flexibilities. While the extent of flexibility is not clear from the FDA’s announcement, the increase in flexibility is likely to have practical implications from an IP perspective.
For clinical development, the FDA reaffirmed that full compliance with current good manufacturing practice (cGMP) requirements (under 21 CFR Part 211) is not expected prior to Phase 2 or Phase 3 trials. The FDA also emphasized that while under 21 CFR 312.23(a)(7), an Investigational New Drug (IND) application must include sufficient chemistry, manufacturing, and controls (CMC) information to ensure the investigational drug’s identity, quality, purity, and strength are appropriate for its stage of development, the final specifications are not expected at earlier stages. Consistent with that framework, the FDA emphasized a lifecycle approach to process and method validation, under which specifications may evolve over time and more flexible release criteria may be used during clinical studies. In practice, this reflects the reality that CGT manufacturing processes can remain fluid well into development, rather than being set in stone early.
The FDA also signaled greater comfort with iterative manufacturing changes as programs advance. The FDA stated that as sponsors move from Phase 1 toward efficacy-focused studies for licensure, it will allow “minor manufacturing changes supported by data showing comparability,” without expecting “overly stringent and onerous” comparability data. The agency also acknowledged that limited lot histories may justify flexibility in setting commercial release specifications. Together, these positions suggest that process improvements made along the way may carry real value, even if they are not part of a final commercial process. For example, a biosimilar may rely on an outdated process initially used for an IND.
Finally, the FDA’s approach to process validation leaves room for multiple manufacturing pathways. For example, by confirming that there is no categorical requirement for three Process Performance Qualification (PPQ) lots and that validation can be tailored to the overall process and available data, the FDA allows for different ways to demonstrate control. The possibility that PPQ lots may be released before all protocol steps are complete further underscores that flexibility, which in turn raises the prospect that a single approved product could be supported by more than one manufacturing approach, each with its own IP implications.
Editor: Brenden S. Gingrich, Ph.D.
[1] https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/flexible-requirements-cell-and-gene-therapies-advance-innovation
[2] https://www.fda.gov/news-events/press-announcements/fda-increases-flexibility-requirements-cell-and-gene-therapies-advance-innovation
[3] https://fortune.com/2026/03/15/australian-tech-entrepreneur-ai-cancer-vaccine-dog-rosie-unsw-mrna/
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