Programs

AVROBIO Pipeline
AVR-RD-01 for Fabry Disease

The aim of AVROBIO’s clinical-stage gene therapy to treat Fabry disease is to deliver lasting and meaningful benefits for patients. The patient’s blood stem cells are extracted and genetically modified by adding a new, fully functional copy of the faulty gene. The modified cells are then delivered back into the patient via a one-time infusion. A durable 24/7 elevation of endogenous enzyme is expected, with the potential to significantly improve patient outcomes and eliminate burdensome and costly lifelong biweekly intravenous infusions of enzyme replacement therapy.

The primary objective of the ongoing Phase 1 trial is to evaluate the safety and toxicity of using the patient’s own stem cells which are genetically modified to express the missing enzyme, alpha galactosidase A (alpha-gal A).

AVR-RD-02 for Gaucher Disease

The aim of AVROBIO’s investigational gene therapy to treat Gaucher disease is to deliver lasting and meaningful benefits for patients. The patient’s blood stem cells are extracted and genetically modified by adding a new, fully functional copy of the faulty gene. The modified cells are then delivered back into the patient via a one-time infusion. A durable 24/7 elevation of endogenous enzyme is expected, with the potential to significantly improve patient outcomes and eliminate burdensome and costly lifelong biweekly intravenous infusions of enzyme replacement therapy.

Preclinical proof-of-concept studies in Gaucher disease mice have demonstrated both prevention in younger mice, and disease reversal in older mice. Investigational New Drug (IND)-enabling studies are now underway in preparation for a Phase 1/2 clinical study.

AVR-ONC-01 for Acute Myeloid Leukemia (AML)

The aim of AVROBIO’s clinical-stage gene therapy to treat AML is to recruit the patient’s own immune system to better detect and eradicate cancer cells. After initial chemotherapy, some of the patient’s residual cancer cells are extracted and genetically modified to express IL-12, a powerful immune system activator. The modified cells are then infused back into the patient via a one-time procedure, with a goal to elicit a potent and durable anti-cancer immune response against remaining AML blasts in the patient’s body.

The primary objective of the ongoing Phase 1 trial is to demonstrate the safety and tolerability of infusing the patient’s AML cells genetically modified to express IL-12 into patients and to determine the maximum tolerated dose.