RegenxBio discloses second serious AE for hypercholesterolemia gene therapy

RegenxBio Inc. (NASDAQ:RGNX) said RGX-501 led to a second treatment-related serious adverse event in a Phase I/II trial to treat homozygous familial hypercholesterolemia (HoFH).

The open-label, international trial has enrolled six patients to receive a single IV infusion of 2.5x10¹² or 7.5x10¹² GC/kg RGX-501. The product is an adeno-associated virus serotype 8 (AAV8) delivering the low-density lipoprotein receptor (LDLR) gene...