Summary
- Liver Therapy Forum weekly digest provides an overview of what's happening in therapeutics for dyslipidemia in 2019. The focus this week is Regenxbio Inc.
- As a leader of AAV-gene based technology, Regenxbio validated its technology with the regulatory approval of Zolgensma, for pediatric patients with spinal muscular atrophy.
- Homozygous familial hypercholesterolemia is an orphan genetic disease characterized by abnormal elevation of LDL-cholesterol that increases risk for cardiovascular diseases.
- Regenxbio has proposed restoring the defective gene using RGX-501 to therapeutically improve abnormal LDL-cholesterol levels.
- Looking for a portfolio of ideas like this one? Members of Liver Therapy Forum (LTF) get exclusive access to our model portfolio. Get started today »
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 delivery platform "that focuses on correcting these defects in genetic diseases by delivering a healthy, working copy of the gene to the cells in need of repair, which potentially enables the body to make the deficient protein. The NAV Technology Platform can also be used to deliver a gene that allows the body to produce a therapeutic protein to treat a specific disease."
