AVROBIO, Inc., a clinical-stage gene therapy company, focuses on developing ex vivo lentiviral-based gene therapies to treat rare diseases following a single dose in the United States. Its gene therapies employ hematopoietic stem cells that are collected from patients and modified with a lentiviral vector to insert functional copies of the gene, which is defective in the target disease. The company's lead product candidate is AVR-RD-01, which is in ongoing Phase II clinical trial and investigator-sponsored Phase I clinical trial for the treatment of Fabry disease. It is also developing AVR-RD-02 that has completed pre-clinical trials for the treatment of type 1 Gaucher disease; AVR-RD-03, which is in preclinical development to treat Pompe disease; and AVR-RD-04 that has completed pre-clinical studies for treating cystinosis. The company was founded in 2015 and is headquartered in Cambridge, Massachusetts.