REGENXBIO has completed dosing of the third cohort in a Phase I clinical trial that seeks to examine RGX-314 for the treatment of patients suffering from wet age-related macular degeneration (wet AMD).

The cohort includes six patients, while topline data from the study is expected to be released later this year.

Go deeper with GlobalData

Go deeper with GlobalData

The gold standard of business intelligence.

Find out more

Discover B2B Marketing That Performs

Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.

Find out more

REGENXBIO chief medical officer Stephen Yoo said: “Completing enrolment of the third cohort in the Phase I clinical trial brings us one step closer toward delivering on the promise of a one-time treatment with rapid and sustained therapeutic effects for patients with wet AMD, one of the largest indications for which gene therapy is being developed.”

The Phase I trial is a multi-centre, open-label, multiple-cohort, dose-escalation study and aims to evaluate the safety and tolerability of RGX-314 as a one-time therapy for patients with previously treated wet AMD.

It intends to enrol about 18 previously treated wet AMD patients who are responsive to anti-vascular endothelial growth factor (anti-VEGF) therapy and are 50 years of age or older.

“Completing enrolment of the third cohort in the Phase I clinical trial brings us one step closer toward delivering a one-time treatment with rapid and sustained therapeutic effects.”

As part of the trial, three doses of RGX-314 will be evaluated.

GlobalData Strategic Intelligence

US Tariffs are shifting - will you react or anticipate?

Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.

By GlobalData

The trial’s primary objective is to examine the safety and tolerability of RGX-314 at 24 weeks after a single dose administered by subretinal delivery.

Its primary endpoints are safety and tolerability, while secondary endpoints are ocular examinations and imaging as well as the need for additional anti-VEGF therapy.

Once the primary study period concludes, the subjects will enter a follow-up period and will continue to be evaluated until week 106 to determine long-term safety and durability of effect of RGX-314.

The trial is being conducted at six retinal surgery centres across the US.

Clinical Trials Arena Excellence Awards - The Benefits of Entering

Gain the recognition you deserve! The Clinical Trials Arena Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving clinical trials industry advancements. Don’t miss your chance to stand out—submit your entry today!

Nominate Now