Aiming for durability

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

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.

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