FDA Announces Reforms to Cell and Gene Therapy Manufacturing Requirements
The FDA issued reforms on January 11, 2026, loosening chemistry, manufacturing, and control requirements for cell and gene therapy products to advance innovation and accommodate the unique complexity of these treatments.
On January 11, 2026, the FDA issued two news releases putting forward reforms to the chemistry, manufacturing, and control requirements for cell and gene therapy products. Under the new FDA policy, the agency has loosened certain requirements given the complexity and individualized nature of gene and cell therapies.
The reforms include changes to clinical development protocols, allowing minor manufacturing changes as sponsors move from phase 1 to studies designed to establish efficacy for licensure. The Center for Biologics Evaluation and Research (CBER) will allow these changes when supported by data showing the comparability of the pre-change and post-change product without expecting overly stringent and onerous comparability data.
For commercial specifications, CBER will consider submissions seeking to re-evaluate and revise product release acceptance criteria based on post-approval manufacturing experience, when manufacturers demonstrate consistent product quality. Additionally, there is no requirement to supply three PPQ lots for process validation.
According to the FDA, over the last decade, the Center for Biologics Evaluation and Research has approved close to 50 cell and gene therapy products. The agency's commissioner described the reforms as "common-sense reforms" to accommodate the unique issues faced by developing cell and gene therapies and is part of the agency's efforts to "remove barriers and perceived misconceptions that stand in the way of expedited product development."
In December 2025, the FDA's Center for Biologics Evaluation and Review approved a gene-based stem cell therapy for Wiskott–Aldrich syndrome, a rare and life-threatening genetic disease. This decision was appropriately based on data from two clinical studies that demonstrated a clinical benefit.
A bibliometric analysis published in the journal Cytotherapy examined over 160,000 papers related to cell and gene therapy published between 1989 and 2023. The analysis revealed that research focusing on hematopoietic stem cell transplantation, including bone marrow transplantation, and ex vivo gene therapy has seen continued growth. In contrast, mesenchymal stem cell therapy and in vivo gene therapy have stagnated in their transition to clinical practice.
The study uncovered variations in research output by region. While Japan reliably contributes a large number of papers in cell therapy, it is lacking in significant qualitative influence. The United States and China lead the field of cell and gene therapy research in both quantity and quality. High-impact papers were often a result of international collaborations, with strong ties between Europe and the United States, as well as strong connections within Europe.