AVROBIO, Inc. is a biotechnology firm in the clinical development stage, dedicated to advancing gene therapies. The company specializes in an ex vivo lentiviral-based approach, aiming to address rare genetic conditions worldwide through a single therapeutic administration. Their innovative methodology involves collecting hematopoietic stem cells directly from patients. These cells are then genetically engineered using a lentiviral vector, which introduces functional copies of the specific gene that is deficient or defective in the patient's condition. Among its pipeline, AVROBIO's lead candidate is AVR-RD-01, designed for the treatment of Fabry disease. The company's therapeutic portfolio also includes AVR-RD-02, currently undergoing Phase 1/2 clinical trials for Type 1 Gaucher disease, alongside AVR-RD-03 for Pompe disease, AVR-RD-04 for cystinosis, and AVR-RD-05 for Hunter syndrome. Additionally, AVR-RD-06 is in preclinical development, targeting Type 3 Gaucher disease. Established in 2015, AVROBIO maintains its corporate headquarters in Cambridge, Massachusetts.
