BioCentury
ARTICLE | Emerging Company Profile

Aiming for durability

Avrobio’s cell therapies have long-term potential in Fabry’s disease, AML

January 20, 2017 10:38 PM UTC

Avrobio Inc. is developing cell therapies that are transduced with lentivirus ex vivo to express therapeutic proteins. The approach could provide long-lasting treatments for patients with monogenic rare diseases or cancer. Data from Phase I programs in Fabry’s disease and acute myelogenous leukemia are expected this year.

Its lead candidate is Fabry’s disease therapy AVR-RD-01, which comprises CD34+ hematopoietic stem cells transduced with a lentivirus ex vivo to express alpha galactosidase A. In Fabry’s, deficiency in this enzyme causes the toxic lipid globotriaosylceramide (GL-3) to accumulate in lysosomes, leading to heart disease and kidney failure...