Gene therapy’s translational problem

Gene therapy is caught in a bind: translational safety issues are disrupting development, with preclinical assays creating red flags where they probably shouldn’t, and failing to detect signs where they probably should.

Commonly deployed preclinical models predict adverse events from adenovirus-associated viral (AAV) vectors that have yet to materialize in any human study. They are serious enough that they can’t be ignored, but they miss other adverse events that do show up in humans. ...