Key points from article :
Regenxbio and AbbVie are developing a gene therapy, ABBV-RGX-314, designed for wet age-related macular degeneration (AMD) to reduce the need for frequent eye injections. Current treatments require regular anti-VEGF injections to manage AMD, but the burden of repeated injections leads to challenges in compliance and potential vision decline over time. ABBV-RGX-314 aims to solve this by using gene therapy to encourage sustained production of therapeutic proteins in retinal cells, potentially offering a one-time solution.
Interim phase 2 data suggest that ABBV-RGX-314 can be delivered safely in-office through a suprachoroidal route, eliminating the need for operating room procedures. Regenxbio used Clearside Biomedical’s technology to deliver the therapy to the suprachoroidal space, making the process feasible in an ophthalmologist’s office. Among 106 patients in the study, no serious drug-related side effects were reported, with only mild inflammation in two cases, both resolved with prophylactic steroid eye drops. This approach is seen as highly acceptable for patients and retina specialists alike.
On the efficacy side, patients showed stable vision (measured by BCVA) and central retinal thickness six months after treatment. Additionally, patients required significantly fewer anti-VEGF injections, with those at the highest dose experiencing an 80% reduction in injection frequency. Notably, half of the patients in this group needed no further injections after the therapy.
Regenxbio views ABBV-RGX-314 as a promising solution for AMD patients who struggle with regular injections. Phase 3 trials for a subretinal delivery version are expected to conclude in 2025, while the next steps for in-office suprachoroidal administration will be determined in collaboration with AbbVie. This therapy holds the potential to simplify treatment and improve patient compliance, which could support its projected multibillion-dollar market value.
