Using Gene Therapy to Create a Drug Biofactory within a Patient

The Bio Report

Wet age-related macular degeneration is a progressive disease and a leading cause of vision loss in patients over the age of 60. Vision loss is caused by the leakage of blood and other fluid from abnormal blood vessel growth underneath and into the retina. Though the condition is treated by a class of therapies known as VEGF inhibitors, these biologics need to be repeatedly injected into patients’ eyes and for a variety of reasons patients’ real-world experience with the drugs don’t match clinical trial outcomes. Adverum Biotechnologies is developing a gene therapy to treat the condition. Rather than addressing an underlying genetic cause though, the gene therapy carries a sequence that causes the eye to produce aflibercept, the VEGF inhibitor marketed as Eyelea and Zaltrap. We spoke to Laurent Fischer, CEO of Adverum, about the company’s gene therapy, how it works, and why the one-and-done approach could translate into better results for patients.