AVROBIO, Inc., a clinical-stage gene therapy company, develops ex vivo lentiviral-based gene therapies to treat rare diseases following a single dose worldwide. 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 that is defective in the target disease. The company's product includes AVR-RD-01, a gene therapy for the treatment of Fabry disease.

It is also developing AVR-RD-02, which is in phase 1/2 clinical trial for the treatment of type 1 Gaucher disease; AVR-RD-03, for the treatment of Pompe disease; AVR-RD-04 for the treatment of cystinosis; AVR-RD-05 for the treatment of Hunter syndrome; and AVR-RD-06 that is in preclinical stage for the treatment of Gaucher disease type 3.