Key information

🔎 Fund Overview

AVROBIO, Inc. is a gene therapy company. The Company is focused on developing potentially curative hematopoietic stem cells (HSC) gene therapies to treat patients with rare diseases following a single dose treatment regimen. The gene therapies that the Company is engaged in developing employ HSCs that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The Company's development focus has been on a group of rare genetic diseases referred to as lysosomal disorders, primarily managed with enzyme replacement therapies (ERTs). Its pipeline is comprised of three HSC gene therapy programs, which include AVR-RD-02 for the treatment of Gaucher disease type 1 and type 3; AVR-RD-03 for the treatment of Pompe disease, and AVR-RD-01 for the treatment of Fabry disease.