The following transcript has been edited for clarity.
Natalia Misciattelli, PhD: Hi, we’re here in New Orleans at Eyecelerator. This is Natalia Misciattelli, PhD, the CEO of AAVantgarde Bio. I’m joined by—
Jayashree Sahni, MD, FRCOphth, PhD, EMBA: —Jayashree Sahni, I’m the chief medical officer of AAVantgarde Bio.
Dr. Misciattelli: We’re super excited to be here and to talk about what we’re doing at AAVantgarde. Last week, we were at EURETINA, where we presented data, and we’re continuing to talk about our data here at the American Academy of Ophthalmology (AAO) and Eyecelerator.
AAVantgarde is an ophthalmology company. We're focused on inherited retinal diseases, and we have 2 programs in the clinic. Our first program is for Usher syndrome type 1B, and our second program is for Stargardt disease. Both of the programs are in the clinic. They’re using a platform technology that allows us to treat the root cause of the disease. That means that we can treat the basis of the disease in genetic diseases where we know what mutation causes the disease. As we've progressed with very, very robust preclinical data into the clinic, we're now starting to see the data being generated, and that's the data that Jayashree will be talking about.
Dr. Sahni: Our first indication, where we are testing our dual AAV DNA-splicing technology, is retinitis pigmentosa caused by mutations in the MYO7A gene in patients with Usher syndrome type 1B. This is a multisensory, devastating disease where the children are born profoundly deaf, and over the course of the next couple of decades, they progressively lose their vision.
We are very pleased to say that we have treated 15 participants in our phase 1/2 study, and the data were presented at EURETINA. We are also very pleased to say that the safety looks favorable, and we have not observed any serious adverse events or dose-limiting toxicities. No patients have withdrawn, and immunosuppression is working very well.
We also have some early signals of efficacy in both BCVA and LLVA, with improvements of 3 or more lines in LLVA. Overall, the data look promising, and at this point, we are looking to develop the program in phase 3.
We also have a second program, which is using the intein technology. We started the program based on very strong preclinical data, and now we are in phase 1/2. The data look promising, and we hope to be in phase 3 with that program late next year.
Dr. Misciattelli: Thank you very much. RP