AbbVie and Regenxbio Share Updates on ABBV-RGX-314 Gene Therapy for Wet AMD and Diabetic Retinopathy

AbbVie and Regenxbio have announced updates on the clinical development of ABBV-RGX-314, a potential one-time gene therapy for wet age-related macular degeneration (AMD), diabetic retinopathy (DR), and other chronic retinal conditions.

Overview of ABBV-RGX-314

ABBV-RGX-314 utilizes the NAV AAV8 vector to deliver an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). By targeting the VEGF pathway, this therapy aims to prevent the growth of leaky blood vessels and reduce fluid accumulation in the retina, addressing key factors in wet age-related macular degeneration and diabetic retinopathy.

Clinical Program Updates

Pivotal Trials in Wet Age-related Macular Degeneration

       • ATMOSPHERE and ASCENT Trials:
The pivotal trials are evaluating the safety and efficacy of subretinal delivery of ABBV-RGX-314 in patients with wet age-related macular degeneration.

       • Data Timeline: Results from these trials are anticipated in 2026.

Phase 3 Program for Suprachoroidal Delivery

       • In-Office SCS Microinjector:
AbbVie and Regenxbio are advancing a phase 3 clinical program to deliver ABBV-RGX-314 to the suprachoroidal space of the eye using the SCS Microinjector.

       • This minimally invasive, in-office procedure could make gene therapy delivery more accessible for patients.

       • The SCS Microinjector technology is licensed exclusively from Clearside Biomedical for delivering gene therapies.

Dual Administration Approaches

The development of ABBV-RGX-314 includes two distinct delivery methods:

       1. Subretinal Delivery: A standardized surgical procedure for precise placement of the gene therapy.

       2. Suprachoroidal Delivery: A less invasive, in-office approach utilizing the SCS Microinjector.

Future Potential

If successful, ABBV-RGX-314 could offer a transformative one-time treatment for wet age-related macular degeneration, diabetic retinopathy, and other retinal diseases, reducing the need for frequent anti-VEGF injections and providing durable vision improvements for patients.

This innovative therapy represents a significant step forward in addressing the burden of chronic retinal conditions, with the potential to improve outcomes and accessibility for millions of patients worldwide.