A single-dose gene therapy for neovascular age-related macular degeneration (nAMD) is now in Phase 3 clinical trials and, if approved, could replace repetitive anti-VEGF intravitreal injections.
While anti-VEGF intravitreal treatments are considered the gold-standard, the treatments are not once-off, with patients requiring frequent, ongoing injections.
This, coupled with the cost of private ophthalmology services, results in a significant dropout rate with around 20% of Australians reported to stop treatment within a year, and 50% within five years.
Gene therapy ABBV-RGX-314, developed by US-based biotech company RegenXBio, delivers anti-VEGF genetic material to the diseased eye in a once-off, subretinal dose.
In Phase 1/2a trial data recently published in The Lancet, the therapy was described to have the potential to “control exudation, maintain vision, and reduce treatment burden after a single administration”.
Forty-two participants were recruited and administered a single subretinal ABBV-RGX-314 injection of varying doses. They were followed for up two years and reported their vision had stabilised or improved by the end of the trial period. Most participants required few or no supplemental anti-VEGF injections.
The therapy demonstrated a favourable safety profile. However, it was possibly related to one adverse event with the participant experiencing severe vision reduction one year after treatment.
“We have started 2024 with strong, positive new data from the ABBV-RGX-314 program, and we believe that there is multi-billion-dollar potential for ABBV-RGX-314 to become a first-in-class gene therapy for wet AMD and the standard of care to treat and prevent progression of diabetic retinopathy,” RegenXBio CEO and president Mr Kenneth Mills said.
“To have these Phase 1/2a data published in The Lancet highlights the groundbreaking work of our scientists and investigators, and further validates the clinically transformative nature of ABBV-RGX-314 as a potential one-time gene therapy for wet AMD that may help patients maintain or improve their vision.”
RegenxBio said enrolment for current clinical trials for the therapy are on track and are expected to support global regulatory submission with the Food and Drug Administration (FDA) and European Medicines Agency (EMA) in late 2025 through the first half of 2026.
More reading
Novartis discontinuing GT005 gene therapy clinical trial for geographic atrophy
Gene therapy trial for dry AMD under way in Australia
TGA approves Luxturna gene therapy
