The IGA are excited to be working in partnership with Avrobio to explore gene therapy as a potential treatment for Gaucher patients.
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.
For more information about AVROBIO's pipeline and technology, please visit their website