At Retinal Physician, we are incredibly excited about this special edition of the magazine that was guest-edited by Drs. Peter Campochiaro, Arshad Khanani, and Rishi Singh on innovations in gene therapy. Starting with the FDA approval of voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics) on December 18, 2017, for patients with RPE65-mutated Leber congenital amaurosis, to the numerous ongoing gene therapy trials in inherited retinal degenerations, optogenetics, and retinal vascular diseases, the field has truly exploded.

Just this month, AGTC announced positive phase 1/2 results using an AAV2 vector delivered subretinally to produce human RPGR to treat X-linked retinitis pigmentosa. In this issue, we explore this exciting field looking at such things as vector delivery (intravitreal, subretinal, suprachoroidal), vector considerations (AAV, lentivirus), and clinical uses. In particular, we explore the many variables that can affect transduction rates and thus the effectiveness of any given gene therapy treatment. With each trial, we learn more about how best to treat retinal diseases and deliver therapeutic proteins. It truly is an exciting time.

We plan to follow up with additional Retinal Physician focus issues with expert guest editors. We hope you enjoy this special edition of Retinal Physician, and we thank REGENXBIO for their support.