We are a clinical stage gene therapy company focused on developing potentially curative ex vivo lentiviral-based gene therapies to treat rare diseases following a single dose. Our gene therapies employ hematopoietic stem cells that are extracted from the patient and then modified with lentiviral vectors to insert a functional copy of the gene that is defective in the target disease. We believe that our approach, which transforms stem cells from patients into therapeutic products, has the potential to provide curative benefit for a range of diseases in an outpatient setting. Our initial focus is on a group of rare genetic diseases referred to as lysosomal storage diseases, which today are primarily managed with enzyme replacement therapies, or ERTs. These lysosomal storage diseases have well understood biologies, identified patient populations and represent large market opportunities with approximately $4.0 billion in worldwide net sales in 2017.
Our initial pipeline is comprised of four lentiviral-based gene therapies, including AVR-RD-01 for the treatment of Fabry disease, AVR-RD-02 for the treatment of Gaucher disease, AVR-RD-03 for the treatment of Pompe disease and AVR-RD-04 for the treatment of cystinosis. AVR-RD-01 is currently being evaluated in a Phase 1 clinical trial to assess safety and toxicity, as well as preliminary efficacy. We expect to initiate our Phase 2 clinical trial of AVR-RD-01 in mid-2018. In addition, Phase 1/2 clinical trials for both AVR-RD-02 and AVR-RD-04 are being planned and we expect patients will be dosed in 2019.