The US Food and Drug Administration (FDA) has accepted an application for the CRISPR genome-editing-based gene therapy to treat blood diseases sickle cell disease and beta-thalassaemia.
The FDA will make a decision by 8 December 2023 and, if approved, the drug will be the first CRISPR therapy to reach the market in the USA.
The drug has been shown to be effective in clinical trials (see BioNews 1108 and 1052).
The FDA will issue separate verdicts on the drug's use in sickle cell disease and beta-thalassemia.
For more information see BioPharmaDive.
