Posted by Michael Wonder on 31 Oct 2023
US FDA awards both rare paediatric disease and orphan drug designations to HG204, a CRISPR RNA editing therapy, for the treatment of MECP2 duplication syndrome
31 October 2023 - HG204 is the world first CRISPR RNA-editing therapy for the treatment of MECP2 duplication syndrome granted both rare paediatric disease designation and orphan drug designation by the US FDA.
HuidaGene Therapeutics today announced the US FDA had granted both the rare paediatric disease designation and orphan drug designation to HG204 for the treatment of MECP2 duplication syndrome, a rare and fatal childhood neurodevelopmental disorder for which there is no treatment.
