The U.S. Food and Drug Administration (FDA) announced new regulatory measures aimed at accelerating the development of cell and gene therapies (CGTs). The agency has decided to ease some of its chemistry, manufacturing, and control requirements for these types of biologics.
In statements issued on Sunday, FDA Commissioner Marty Makary described the changes as “common-sense reforms” that recognize the complexity of CGTs. These therapies are often tailored to individual patients or produced in small quantities. The Center for Biologics Evaluation and Research has agreed to relax certain standards “that accommodate the unique characteristics of these innovative therapies.”
One notable adjustment is that manufacturers will not have to meet specific manufacturing specifications for biologics as outlined in Chapter 600 of the Code of Federal Regulations when an investigational gene therapy progresses beyond Phase I clinical trials. Normally, these standards apply once a drug candidate enters Phase II or III.
The FDA also indicated it would be flexible regarding minor changes in manufacturing processes during later stages of product development, provided companies can show that such modifications do not substantially alter the therapy.
Additionally, regulators expressed willingness to consider adjustments to a product’s commercial specifications.
Despite introducing more flexibility, the agency emphasized its continued commitment to quality. It stated that regulatory oversight will still uphold “rigorous quality standards through appropriate control measures.” The agency added, “The new flexibilities will enable progress while not compromising or undermining the FDA’s ability to assure safety, purity and potency of a product.”
These changes follow other recent policy updates intended to help CGT developers. In November last year, the FDA introduced its plausible mechanism pathway to support treatments targeting diseases with limited patient populations—many being CGTs. This approach allows regulators to consider both disease biology and how an experimental therapy addresses it.
Earlier in October 2025, draft guidance documents were released proposing streamlined review procedures for CGTs. Among these was approval for self-controlled trials using each patient’s baseline as a reference point for measuring effectiveness.
