Eye On the Cure Research News

Jan 7, 2021

REGENXBIO’s Wet AMD Gene Therapy Moving into Phase 3 Trials

In a Phase ½ clinical trial, the emerging treatment reduced the burden of regular anti-VEGF injections

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REGENXBIO, a clinical-stage gene therapy biotechnology company, is planning two pivotal, Phase 3 clinical trials for RGX-314, its gene therapy designed to halt the growth of leaky blood vessels that cause retinal degeneration and central vision loss in people with the wet form of age-related macular degeneration (AMD). Dosing in the first trial, known as ATMOSPHERE™, is expected to begin in the first quarter of 2021. The second trial is expected to begin in the second half of 2021.

Though there are FDA-approved treatments for wet age-related macular degeneration (AMD) such as Lucentis™ (ranibizumab) and Eylea® (aflibercept), these therapies, known as anti-VEGFs, need to be injected into the eye at a doctor’s office for the rest of the patient’s life. In some cases, the injections are given monthly. In other cases, the anti-VEGFs are administered on an as needed basis. Regardless, the never-ending injections are neither pleasant nor convenient for the patient.

REGENXBIO’s emerging gene therapy is designed to greatly reduce the need for repetitive anti-VEGF injections. RGX-314 is administered as a one-time subretinal injection in the affected eye and provides sustained production of the anti-VEGF protein.

Participants in the Phase 1/2 trial for RGX-314 had a significant reduction in their anti-VEGF treatment burden.

REGENXBIO is also conducting a Phase 2 clinical trial, known as AAVIATE™, for RGX-314 administered through suprachoroidal delivery, an injection underneath the sclera, the white outer layer of the eye. Unlike subretinal injections, which are surgical procedures, suprachoroidal injections are usually performed in an eye doctor’s office.