AVROBIO, Inc. is a clinical-stage biotechnology company developing lentiviral-based gene therapies for rare lysosomal storage disorders and other genetic diseases. Its lead program, AVR-RD-01, is a lentiviral gene therapy in Phase 1 for Fabry disease that produced normal plasma α-galactosidase A activity in the first treated patient at six months. The company’s proprietary 3rd-generation lentiviral platform is designed for stable genomic integration and permanent gene addition in CD34+ stem cells. AVROBIO plans to initiate a Phase 2 trial of AVR-RD-01 this year and to move gene therapy candidates for cystinosis and Gaucher disease into clinical development by mid-2019, and also intends to apply its platform to other diseases including Pompe disease. The company is headquartered in Cambridge, MA and has offices in Toronto, ON. AVROBIO completed a $60 million Series B financing to advance multiple programs and build its lentiviral platform capabilities. AVROBIO is a clinical-stage biotechnology company developing lentiviral-based, ex‑vivo gene therapies for rare diseases and cancer. Its initial clinical programs are Phase 1 candidates for Fabry disease and acute myeloid leukemia (AML). The Fabry program modifies patients' stem cells to add a functional copy of the faulty gene and reinfuses them in a one‑time procedure to produce durable enzyme elevation. The AML program seeks to genetically modify residual patient cells to express IL‑12 after chemotherapy to elicit a durable anti‑cancer immune response. AVROBIO emphasizes single‑infusion, potentially disease‑modifying therapies and is actively building a broader pipeline targeting other rare diseases and solid and liquid cancers. The company is headquartered in Cambridge, MA and has offices in Toronto, ON. It raised new funding to accelerate its clinical programs and pipeline expansion.

Total raised

$85M

Funding rounds

2

Latest round

Series B

Latest activity

Feb 2018