Gene therapy experiments don’t need special regulations, health officials say

Health officials are planning to do away with specific regulations for gene therapy experiments, a sign of the field’s rapid evolution. In 2017, the FDA approved the first three gene therapy products — which use DNA to treat or prevent disease — for use in the U.S. For years, a special NIH advisory panel reviewed applications for gene therapy studies. But in 2016, the NIH scaled back the panel's work and now, wants to shift it to an advisory role. Under proposed changes, the FDA would evaluate gene therapy studies the same way it vets other potential treatments. The public will be able to comment on the proposal soon.

In a commentary just published in NEJM, FDA Commissioner Scott Gottlieb and NIH Director Francis Collins say that as gene therapy advances, the federal framework that oversees it needs to change. "It seems reasonable to envision a day when gene therapy will be a mainstay of treatment for many diseases," they write.