Gene Therapy for Neovascular AMD
PracticeUpdate: What drug is under investigation in the AAVIATE study, and how does it work?
Subretinal injection vs suprachoroidal delivery
Dr. Kondapalli: The AAVIATE study looked at RGX-314, which is a one-time delivery of gene therapy. That’s a monoclonal antibody fragment that would actually express your own anti-VEGF. Sounds sci-fi; but, essentially, the molecule gets placed into the eye, whether that is through a subretinal injection or suprachoroidal delivery, and it's able to generate its own anti-VEGF molecule. Which specific molecule is determined based on the original monoclonal antibody fragment, but that is what the medication is. In particular, the AAVIATE study is looking at suprachoroidal delivery of the medication. And, in this procedure, it's usually traditionally done in the office by a special suprachoroidal injector, and it can provide medication into the eye and differently, or in a different pathway, than a traditional intravitreal injection, which is done also in the office, or subretinal injection that is done usually in the operating room.
Efficacy backed up by data; delivery method in question
PracticeUpdate: Within the context of AAVIATE and given interim results of the trial, how promising is gene therapy for the treatment of neovascular AMD?
Dr. Kondapalli: In the AAVIATE study — this is a phase II clinical trial — what they look at is varying doses of this RGX-314 medication. We want to evaluate the reduction in anti-VEGF treatment burden —so, if patients needed to be rescued, for example, with an anti-VEGF medication in the office — but they also looked at best-corrected visual acuity and central retinal thickness.
What was found in the trial was that suprachoroidal delivery of RGX-314 seems to be an option in those patients who need to have long-term VEGF suppression. Further phase III clinical trials will be pending to look at safety; but, what's interesting, is that RGX-314 is the same exact drug that’s used in two other studies, ATMOSPHERE and ASCENT. However, this was done through subretinal delivery. So, we already have data on the efficacy of the RGX-314; however, we’re just looking at two different methods in terms of getting it into the eye.
PracticeUpdate: Are there other gene therapies in the pipeline for the same indication?
Dr. Kondapalli: Another molecule is ADVM-022. It’s another gene therapy, which is currently in a phase II trial, looking at treatment to suppress VEGF in those patients with wet macular degeneration.
Click on any of these tags to subscribe to Topic Alerts. Once subscribed, you can get a single, daily email any time PracticeUpdate publishes content on the topics that interest you.
Visit your Preferences and Settings section to Manage All Topic Alerts
Additional Info
Disclosure statements are available on the authors' profiles: