Gene Therapy: What To Expect Now And In The Future

A trickle becoming a stream might describe the state of gene therapy development in the US, where in the year 2023 regulators anticipate approving as many gene therapies as they had from 2017-2022. In the process, the US Food and Drug Administration will have unleashed not only viral vector-based gene therapies for ultra-rare diseases, but treatments using newer modalities, like gene editing, to treat more common conditions like sickle cell disease (SCD).