RegenxBio, a publicly-traded biotech firm, released data this week from a Phase 2 clinical trial designed to test its leading genetic therapy product in patients with bilateral wet age-related macular degeneration (AMD). AMD is characterized by abnormal growth of blood vessels in the retina, and is a leading cause of loss of vision in elderly populations globally.
ABBV-RGX-314, developed in collaboration with AbbVie, offers the potential of a one-time treatment for wet AMD and other retinal conditions, including diabetic retinopathy. This is in contrast to existing treatments which rely on repeated intraocular injections of drugs that inhibit a protein known as Vascular Endothelial Growth Factor (VEGF), a protein responsible for the formation of new retinal blood vessels.
The ABBV-RGX-314 therapy is based on a an AAV8 viral vector as a delivery system. The AAV8 platform has been genetically engineered to encode an antibody that can inhibit VEGF for the long-term.
Patients enrolled in the ABBV-RGX-314 Phase 2 sub-study have enjoyed marked benefits after nine months; researchers cite a 97% reduction in the need for anti-VEGF injections, with patients exhibiting proper levels of Best-Corrected Visual Acuity (BCVA) and central retinal thickness following the treatment.
The eye is termed as an immune-privileged part of human anatomy; the inner part of the eye does not contain lymph vessels which ferry White Blood Cells (WBCs) that fight infections, although they can be found in the eye’s blood vessels. The development of viral-based delivery systems for genetic therapy has been greatly hampered by human immunity, since WBCs quickly develop antibodies that identify and attack the viral vector used for the therapy, rendering it ineffective for more than one use. Several companies have therefore developed viral-based delivery systems for therapies that treat AMD and other conditions that affect the retina and other parts of the eye, which uniquely allows for testing these types of therapies in a site where they may exhibit greater efficacy.
Source: REGENXBIO Presents Positive Data from the Phase II Study of Subretinal ABBV-RGX-314 in Patients with Bilateral Wet AMD at AAO 2024
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