BiopharmaDive: Gene therapy approvals bring validation as field closes year on high

In the last four months, the Food and Drug Administration approved more gene therapies for inherited diseases than it had in the five years previous, a flurry of activity that’s lifted a beleaguered sector of the biotechnology industry.

In August and September, the regulator cleared treatments for a severe form of the blood condition beta thalassemia as well as for a childhood brain disease, both developed by Bluebird bio. And in November, partners CSL and UniQure won an FDA OK for Hemgenix, their therapy for hemophilia B.

All three are the product of many years of research. In developing them, their makers have played a large role in pushing the gene therapy field from promising proof-of-concept experiments to convincing late-stage clinical trials.