Federal health officials on Monday laid out a proposal to spur development of customized treatments for patients with hard-to-treat diseases, including for rare genetic conditions that the pharmaceutical industry has long considered unprofitable.
The preliminary Food and Drug Administration guidelines, if implemented, would create a new pathway for bespoke therapies that have only been tested in a handful of patients due to the challenges of conducting larger studies. The FDA announcement specifically mentions gene editing, although agency officials said the new approach could also be used by other drugs and therapies.
