REGENXBIO's Lancet-Published Study Assesses One-Time Gene Therapy ABBV-RGX-314 for Wet AMD

The innovative gene therapy ABBV-RGX-314 is poised to set a new benchmark in the treatment of wet age-related macular degeneration (AMD), offering a sustainable solution to the ongoing vision health challenges associated with chronic anti-VEGF injections. Recent clinical findings, published in The Lancet, have shown that patients treated with ABBV-RGX-314 experienced stable or improved vision and retinal structure for up to two years, with extended data indicating a lasting effect that endures for as long as four years.

REGENXBIO Inc., the biotech firm spearheading the development of this potential breakthrough therapy, is optimistic about the prospects of ABBV-RGX-314, which is currently undergoing pivotal trials. The company's CEO, Kenneth T. Mills, has expressed confidence in the therapy's billion-dollar potential, not only for wet AMD but also for treating and preventing the progression of diabetic retinopathy.

The ABBV-RGX-314 treatment, developed in collaboration with AbbVie, is a one-time gene therapy that utilizes the NAV® AAV8 vector to deliver an antibody fragment capable of inhibiting vascular endothelial growth factor (VEGF). This approach is designed to obstruct the VEGF pathway, which is responsible for the growth of abnormal blood vessels that lead to fluid accumulation in the retina.

The Phase I/IIa trial results, which were well-received, have paved the way for ongoing pivotal trials known as ATMOSPHERE® and ASCENT™. These trials are progressing as planned, with the anticipation of supporting global regulatory submissions to both the U.S. Food and Drug Administration and the European Medicines Agency by late 2025 or the first half of 2026.

Wet AMD, a leading cause of vision loss, is characterized by the formation of new, leaky blood vessels in the retina. Despite the current standard of care involving frequent anti-VEGF injections, patient adherence is often hindered by the treatment's burden, leading to progressive vision loss. The introduction of ABBV-RGX-314 could offer a transformative, long-lasting treatment alternative.

REGENXBIO, established in 2009, is a frontrunner in the field of gene therapy, with a focus on AAV Therapeutics. The company's pipeline includes treatments for various retinal and rare diseases, leveraging the potential of one-time treatments to revolutionize healthcare delivery for millions. Their AAV Therapeutic platform has already been utilized to treat thousands of patients, including those administered Novartis' ZOLGENSMA for spinal muscular atrophy.

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