The following transcript has been edited for clarity. 

Hi everyone, I’m Aaron Nagiel, MD, PhD. I’m from Children’s Hospital Los Angeles, affiliated with USC Eye Institute. I’m really excited to be here in Ushuaia, where I presented on two interesting topics related to gene therapy. As you know, this is a rapidly evolving field and it’s interesting that I’m speaking both about Luxturna (voretigene neparvovec-rzyl; Spark Therapeutics), which is our first approved gene therapy, and also one of our newest gene therapies by Opus Genetics for BEST1-related retinal diseases.

Luxturna, as we know, has been a great success. Across the world we’ve treated hundreds of patients with this gene therapy. And what we’ve seen in our post-authorization safety data in the real world is that it really does behave as we thought in the clinical trials. One aspect that wasn’t really found [in trials] was this issue of chorioretinal atrophy, which we described and have since published on many times. And it does give us reason to think that perhaps certain vectors and in the right setting could cause degeneration of the photoreceptors and retinal pigment epithelium (RPE). But so far, this hasn’t really limited the benefits that the patients feel.

Moving to one of the newest gene therapies, it’s really exciting to talk about Opus Genetics’s OPGx-BEST1 gene therapy for both autosomal recessive and dominant forms. We were one of those centers for that study, and I believe this is the first time the data is being presented at an international conference. What we found in these five patients is that the gene therapy was very well tolerated with really no major safety issues. There were some interesting signs of potential efficacy, which we hope to look into more in cohort two, which is going to have a higher dose and even more patients treated. RP