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).

The most recent addition to the armamentarium is BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec), which the FDA approved for hemophilia A on 29 June after a long and winding road to market. (Also see "Its Gene Therapy Roctavian Finally Approved With $2.9m Price, BioMarin Aims To Prove Doubters Wrong" - Scrip, 30 June, 2023.) Just seven days before, the agency gave the green light to Sarepta Therapeutics, Inc