The FDA has published rules for tailored gene therapies and expects a large number of applications for approval for rare diseases through a new "likely pathway of mechanism" [original content]. On 11 January 2026, it announced a more flexible approach to chemistry, manufacturing and control (CMC) requirements for cell and gene therapies (CGT), speeding up product development before a Biologics License Application (BLA)[2][4]. Over the past decade, CBER has approved nearly 50 CGTs targeting serious or life-threatening conditions with unmet medical need[2]. Flexibility includes less stringent quality release criteria in investigative studies, allowing minor changes in production with comparability data, and modifying specifications based on post-regulatory production[4]. These changes maintain the safety, purity and effectiveness of the products without compromise[2][4]. FDA Commissioner Marty Makary stated that regulatory flexibility is tailored to the unique characteristics of CGT to promote innovation[2].