CBER Director Discusses Regulation of Human Genome Editing

With more than a dozen companies developing medicine that use gene editing technologies, the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) Director Peter Marks discussed the risk-based approach his center is taking.

While noting the huge potential for gene editing modifications or corrections in medicine, Marks also discussed in the National Academy of Medicine webinar how FDA cannot currently accept applications for clinical trials related to editing parts of the genome that can be passed down to subsequent generations.





Marks also noted that FDA has the advantage of being able to look across the gene editing clinical trial landscape and spot any safety issues that may be common to ensure “trials are as safe as they possibly can be.”

However, Stanford University professor Matthew Porteus noted that although FDA can see the effects from a range of trials, the agency cannot inform the public on what it sees because of proprietary information, which Porteus said needs to be part of an ongoing conversation.

And in response to a question on “bio-hacking,” which has garnered media attention recently, Marks added, “If we don’t use gene editing carefully, we can set back the field by a decade or more, much in the same way as what happened in 1999,” when the first person publicly identified as having died in a clinical trial for gene therapy.

Sangamo Therapeutics CEO Sandy Macrae, whose company on Thursday also signed off on a potentially multi-billion-dollar research deal with Gilead, noted gene editing targets, novel delivery technologies and companies working to develop such medicines.