The U.S. has seen the approval of numerous cell and gene therapies in the past decade, promising long-term benefits for severe diseases. Peter Marks, who led the FDA’s Center for Biologics Evaluation and Research (CBER) since 2016, played a pivotal role in these approvals, overseeing the clearance of groundbreaking treatments including the first gene therapy, CAR-T cell therapy, and CRISPR gene editing medicine. He advocated for flexible review processes, which, while praised, also drew criticism.
Marks resigned Friday, leaving cell and gene therapy developers without a key supporter at the FDA. This departure adds uncertainty to an industry already facing investment challenges.
Stephan Grupp, head of cell therapy and transplant at Children’s Hospital of Philadelphia, described Marks as a “scientific and regulatory giant,” crediting him with facilitating progress in the field while maintaining safety standards. Katherine High, a prominent gene therapy researcher, highlighted Marks’ “vision, scientific rigor, and outstanding clinical judgment,” noting his efforts in building a regulatory infrastructure that accelerated approvals, including for gene editing.
Cell and gene therapy development faces unique hurdles compared to traditional drugmaking. These therapies are complex, involving cell or genetic manipulation and specialized delivery methods. Establishing safety and efficacy has necessitated new regulatory frameworks and manufacturing processes.
https://www.fiercepharma.com/pharma/after-patient-death-sarepta-and-roche-pause-3-trials-gene-therapy-elevidys-europe
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