ASRS 2026: 4D-150 gene therapy shows sustained treatment-burden reduction in wet AMD at 2 years

Two-year data points to reduced injection burden

Carl C. Awh, MD, FASRS, president of Tennessee Retina in Nashville, Tennessee, presented late-breaking 2-year outcomes for 4D-150 (4D Molecular Therapeutics), an investigational intravitreal gene therapy, at the American Society of Retina Specialists (ASRS) 2026 annual meeting in Montreal. Awh reported data from patients with more moderate neovascular age-related macular degeneration (AMD), a population he said better reflects patients typically seen in clinical practice than the more severe and recalcitrant cases also studied in the program.

Among patients with disease duration under 2 years, a single injection of 4D-150 produced a substantial reduction in treatment burden. Approximately half of these patients did not require another injection over 2 years while maintaining vision and anatomy, and 67% to 69% needed 2 or fewer injections during that period. In a subgroup with more recently diagnosed disease—average duration under 3 months—the reduction in estimated treatment burden reached 87%. In this subgroup, 87% of patients went 2 years without an additional injection, and the large majority required fewer than 2 injections over 2 years.

Favorable safety profile

Awh said the efficacy results were paired with reassuring safety data across all patients followed with 4D-150, with follow-up now extending to 4 years in some patients. Only 2 eyes experienced mild irritation within the first 28 weeks after injection, managed with topical therapy alone; no other eyes experienced serious adverse events.

Looking to phase 3 and beyond

Awh said he hopes phase 3 trials mirror the phase 2 results, with sustained vision maintenance, minimal additional treatment, and a comparable safety profile. He noted that other gene therapies have shown potentially permanent or long-lasting effects, and that maintaining results at each successive follow-up interval has been reassuring. He pointed to undertreatment as a common problem with standard anti-VEGF injection schedules, given the difficulty of returning for frequent, precisely timed injections, and said a sustained backbone therapy—supplemented as needed in some eyes—could lead to better long-term vision outcomes for patients.

Awh said gene therapy is among the most compelling options on the horizon for retina specialists, particularly because it can be delivered using an injection technique already familiar to physicians. He added that it remains premature to compare gene therapies against one another until more agents are approved, but said he welcomes having additional treatment options for patients.

Reference:

1. Awh CC. Presented at: American Society of Retina Specialists (ASRS) 2026 Annual Meeting; July 15-18, 2026; Montreal, Quebec, Canada.