Subject:Exciting AVROBIO update on the development of gene therapies for Fabry disease
Date: 04/09/2019
Time: 03:13 PM
Body:AVROBIO, a leader in lentiviral-based gene therapies, is a clinical stage company developing disruptive therapies that have the potential to transform patients’ lives in a single dose.

Gene therapy involves the transplantation of normal genes into cells in order to correct genetic disorders. Gene therapy uses a vector (see illustration below) to carry and deliver a working copy of a gene to the body’s cells. A vector is a deactivated virus that is unable to reproduce and spread (#1 below). There are different types of vectors. AVROBIO’s
plato™ is a gene therapy platform which uses a lentiviral vector and is designed to target underlying genetic disease by restoring normal gene function (#2 below) and enzyme production.

Fabry Support & Information Group