The FDA has approved two gene therapies for inherited diseases: the blindness treatment Luxturna in 2017 and the neuromuscular disease therapy Zolgensma in 2019.
Since then, despite a boom in gene therapy research among biotech and pharma companies, safety hurdles in clinical trials and a seeming shift in the FDA’s thinking have slowed development somewhat. In 2020, for instance, the FDA rejected another hemophilia gene therapy from BioMarin Pharmaceutical, requesting additional data the drugmaker claimed wasn’t originally required.
