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.

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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.

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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.

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