AVROBIO is a clinical-stage gene therapy company that creates ex vivo lentiviral-based gene therapies for rare diseases globally, given in a single dose. They modify hematopoietic stem cells using a lentiviral vector, inserting functional copies that target the specific disease. Their product includes AVR-RD-01, a gene therapy that treats Fabry disease, and they are developing five additional treatments for different diseases, including Gaucher, Pompe, cystinosis, and Hunter syndrome.