Company to view next
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 lead product candidate is AVR-RD-01, a gene therapy which is in ongoing company-sponsored Phase 2 clinical trial and investigator-sponsored Phase 1 clinical trial 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, which is in preclinical development to treat Pompe disease; AVR-RD-04 that is in investigator-sponsored Phase 1/2 clinical trial to treat cystinosis; AVR-RD-05 for the treatment of Hunter syndrome; and AVR-RD-06 that is in preclinical stage to treat Gaucher disease type 3. The company was incorporated in 2015 and is headquartered in Cambridge, Massachusetts.