Gene Therapy in Neovascular AMD and DME


Drs Nathan Steinle, Adrienne Scott, Carl Regillo, and Prethy Rao highlight gene therapy as a future treatment of neovascular AMD and DME and how it will significantly reduce the burden on patients.

Nathan Steinle, MD: How about if we switch over to gene therapy? I’ll open this to everyone. It’s really fascinating. We’re talking about all these injections but maybe we’re going to stop these injections someday. What are your thoughts in general about gene therapy and how the field is evolving over time?

Prethy Rao, MD, MPH: I am super excited about it.

Nathan Steinle, MD: I bet.

Prethy Rao, MD, MPH: I think in the era of treatment burden and with the dry-emerging therapies for dry AMD [age-related macular degeneration], being able to say, “OK, you can maybe get 1 or 2 a year,” will change the lifestyle of the patient in terms of being able to get to the appointment. So, I’m super excited about it. I’m a little hesitant in terms of more of the inflammatory effect from these injections. A lot of these trials are now adjusting and adding steroids on top of their injections, which I think makes me feel a little bit better about having the inflammation afterward.

Nathan Steinle, MD: Sure.

Adrienne Scott, MD: Yes. I agree completely. I think gene therapy treatments are the game changers that we really want to be able to offer our patients. I think the jury is still out about the mechanism of administration. If it’s subretinal, is it suprachoroidal or is it intravitreal? Probably not. We don’t know. But I think that’s just going to be the game changer we’re all looking for.

Carl Regillo, MD, FACS, FASRS: Yes. It’s an attractive notion. I’m super keen on the notion of sustained delivery, getting good long-term vision outcomes with continuous delivery of a VEGF-A blocker. Because of all the gene therapy programs—there are 3 now, 2 of which are intravitreal, and the other product is either suprachoroidal or subretinal—are aiming to produce an anti-VEGF-A drug. And we know that works great and continuously. We know from the port delivery system approach long-term vision outcomes are fantastic for something that can give us adequate levels continuously of a VEGF-A blocker.

And so, proof of principles there with sustained delivery, gene therapy holds promise. I think it’s very attractive for our patients, especially if it’s easy to administer and inflammation doesn’t prove to be an issue. Because it is something that we have to contend with, address, treat, suppress, and so forth in at least most of the approaches. Maybe not subretinal but suprachoroidal and intravitreal, some degree of upfront corticosteroid use is necessary to suppress inflammation, which is to be expected with the administration of a viral vector. It is inflammatory in nature, at least upfront. But we also have the potential for another sustained delivery which are the TKI [tyrosine kinase inhibitor] programs and there’s 3 active ones there.

Transcript edited for clarity

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