
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. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.
AVROBIO was founded in 2014 as a biotechnology company with a core focus on gene therapies. Over the years, the company has grown from a small start-up to a leader in lentiviral-based gene therapies. Today, AVROBIO is a clinical-stage public biotechnology company with a deep commitment to transforming the lives of patients living with serious diseases through the advancement of groundbreaking gene therapies.
We are committed to accelerating the development of curative gene therapies to transform the lives of patients with serious diseases.
Our vision is to become the leader in developing and commercializing cutting-edge gene therapy treatments for serious diseases.