The following transcript has been edited for clarity.

Diana V. Do, MD: Hi, I'm with my friend and colleague, Anat Loewenstein, MD, MHA. You presented some very exciting data today on gene therapy for diabetic macular edema (DME). Can you give us a quick summary?

Anat Loewenstein, MD, MHA: Thank you very much, Diana. It’s a pleasure to be here with you.

My talk was about the SPECTRA trial, the phase 2 trial for diabetic macular edema for 4D-150 (4D Molecular Therapeutics). I think it's a very exciting trial. This trial was aiming mainly to look at safety and dose determination. What it actually showed is, first of all, that the drug is extremely safe. There were no intraocular inflammation (IOI) events. Patients received 16-week prophylaxis therapy with Durezol (difluprednate ophthalmic emulsion 0.05%; Alcon). However, no one needed to modify the treatment and everything went very well and there was no IOI or vasculitis—nothing whatsoever. In addition, there was a pretty nice dose response, where the visual acuity was increased and maintained and achieved a 9.7 letter increase in the high dose. This was paralleled by a nice decrease in central subfield thickness that also showed a dose response.

So overall, very good data. And this is with dual transgene that actually has a payload of both aflibercept and an RNAi molecule targeting VEGF-C. And together probably this inhibition is really good. It has the R100 capsid, which is specifically designed not to incite an inflammatory reaction, and it works.

Dr. Do: It’s amazing because it’s an in-office procedure via intravitreal injection.

Dr. Loewenstein: Yes, I think this is the most important thing. I think that at the end of the day, if we want to go on a large scale, I think that the intravitreal mode of delivery is a winner.

Dr. Do: Well, thank you so much for your leadership and those insights, and we look forward to more updates in the future.

Dr. Loewenstein: Thank you very much, Diana. Thank you for the opportunity. RP