Efficacy and Safety of an AAV-RPE65 Treatment

July 18th, 2017

The RPE65 gene therapy study group at the Wynn Institute for Vision Research at the University of Iowa and The Center for Molecular and Children’s Hospital of Philadelphia published a paper in the journal, The Lancet, which showed the safety and effectiveness of voretigene naparvovec (the "new" name of AAV-RPE65 ) in a phase 3 FDA trial. This is an important milestone for gene therapy. As Dr. Stone recently published in the journal Ophthalmology, most inherited retinal degeneration genes will fit into the adeno-associated viral vector tested in this study. Patients that have other retinal genetic diseases may benefit from the results of this study.

The complete article is available at http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(17)31868-8/fulltext