Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies

Though enzyme replacement therapies have proven a viable strategy for treating lysosomal storage disorders, one problem is that these medicines face challenges reaching all of the cells throughout the body that are affected by these conditions, particularly in the brain. Avrobio is developing one-and-done gene therapies to treat cystinosis and other lysosomal storage disorders to overcome the limits of ERTs and possibly halt or reverse diseases. We spoke to Geoff MacKay, president and CEO of Avrobio, about cystinosis, the company’s gene therapy platform, and how it's leveraging its technology to develop therapies across a range of rare diseases.