Regulators in the USA have paused two experimental gene therapy programmes from biotech company Regenxbio after a serious safety concern emerged.
The US Food and Drug Administration (FDA) imposed a clinical hold on both the RGX‑111 therapy for Hurler syndrome and RGX‑121 for Hunter syndrome following detection of a brain tumour in a child treated with RGX‑111 several years earlier.
The FDA extended the hold to RGX‑121 because of similarities in the therapy designs, even though no similar tumours have been reported in its study participants.
The decision came just weeks before an expected approval decision for RGX‑121.
Read Fierce Biotech for more information.