AVROBIO, Inc., a leader in lentiviral-based gene therapies, is a clinical stage company developing disruptive therapies that have the potential to transform patients’ lives in a single dose.
Our focus is on serious rare diseases, where a faulty gene results in a lack of an essential enzyme or protein. We plan to initiate a Phase 1/2 trial to evaluate the safety and efficacy of AVR-RD-02 in patients with Type I Gaucher Disease. In this planned trial, we will evaluate the potential for patients’ own genetically modified stem cells to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher Disease.
AVROBIO also aims to understand and support the journey and unique needs of patients and their families. We value the vital work of patient organizations and seek to build partnerships towards our common goals.
AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit www.avrobio.com.