PRVL - Regenxbio And RGX-501 In Homozygous Familial Hypercholesterolemia
Introduction
In Q2/2019, Regenxbio Inc. (RGNX), achieved its first commercial-stage success with FDA approval of Zolgensma, a gene therapy infusion therapy for pediatric patients with spinal muscular atrophy ((SMA)), a leading genetic cause of infant mortality.
RGNX is a small-cap ($1.42B) pioneer of adeno-associated virus ((AAV)) gene-based therapeutics for the potential treatment of diverse diseases including Wet age-related macular degeneration, Mucopolysaccharidosis Type II, diabetic retinopathy and Homozygous familial hypercholesterolemia (HoFH), the focus of this article.
Its clinical approach is based on the NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene