Dilsher Dhoot, MD, on a Promising Start With Diabetic Retinopathy Gene Therapy RGX-314


The adjunct clinical assistant professor, ophthalmology, Keck School of Medicine, University of Southern California discussed new data from the ALTITTUDE trial.

“There's still so many questions that remain, looking at long term gene expression and safety data is going to be key. I'm excited to see, at this higher dose level, how our patients do in terms of efficacy, and top line results also. So, I think there certainly still questions that remain. But you know, the data to date is very encouraging. And I really feel that this is one area in ophthalmology and retina that is very exciting.”

RGX-314 (REGENXBIO) gene therapy, delivered with a one-time suprachoroidal injection, was well-tolerated and yielded improvements in diabetic retinopathy severity scale (DRSS) scores in patients with DR treated in the phase 2 ALTITUDE trial (NCT04567550). Updated data from the study were presented by Dilsher S. Dhoot, MD, ophthalmologist, California Retina Consultants, and adjunct clinical assistant professor, ophthalmology, Keck School of Medicine, University of Southern California, at the American Society of Retina Specialists (ASRS) 2023 Annual Meeting, held July 29-August 1 in Seattle, Washington.

Dhoot presented positive preliminary findings which included no serious adverse events related to RGX-314, stable best-corrected visual acuity through 6 months, and improvements in DRSS, including a 2-step improvement in 20% of treated participants compared to 10% of control participants. Additionally, there were no cases of intraocular inflammation in a new cohort using an additional prophylactic steroid eye drop regimen.

CGTLive spoke with Dhoot to learn more about the data presented at ASRS. He went over the design of the study and the encouraging safety and efficacy findings observed.

Dhoot DS. Suprachoroidal delivery of investigational ABBV-RGX-314 for diabetic retinopathy: The phase II ALTITUDE® study. Presented at: ASRS 2023 Annual Meeting, July 28-August 1; Seattle, Washington.
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