EURETINA 2026: 3 gene therapy programmes for nAMD approaching phase 3 readouts

At the 26th EURETINA Congress in Vienna, Arshad Khanani, MD, FASRS, of Sierra Eye Associates, presented an update on gene therapy for neovascular age-related macular degeneration (nAMD) during an invited EURETINA session. He reviewed 3 programmes in pivotal phase 3 development, all of which are expected to read out over the next 12 months.

Subretinal delivery: sura-vec

The first programme is surabgene lomparvovec (sura-vec; AbbVie and REGENXBIO), a gene therapy delivered subretinally. According to Khanani, its phase 3 trials, ATMOSPHERE and ASCENT, are expected to read out by the end of 2026.

Intravitreal delivery: 4D-150

The second, 4D-150 (4D Molecular Therapeutics), is an intravitreal gene therapy with a dual mechanism of action. It drives secretion of aflibercept protein and also delivers RNA interference targeting VEGF-C. Khanani noted that the phase 1/2 PRISM study produced positive data. The phase 3 trials, 4FRONT-1 and 4FRONT-2, are now fully enrolled, and he expects results in the first and second halves of 2027.

Lessons from ixo-vec

The third programme, ixoberogene soroparvovec (ixo-vec; Adverum Biotechnologies), has been in development for a long time. Khanani said its phase 1 study informed much of the field's understanding of the risks and benefits of gene therapy. He presented a summary of the OPTIC and LUNA data. Ixo-vec is now in phase 3, with 1 trial fully enrolled and the other still enrolling.

A potential paradigm shift

“The bottom line is that gene therapy for neovascular AMD is close to being a reality for our patients,” Khanani said.

He described what an effective and safe one-time gene therapy injection could offer. Between 15% and 60% of patients might need no further treatment. Those who still require treatment might move from injections every 1 to 2 months to injections every 6 or 9 months. Khanani said this would represent a paradigm shift for patients with nAMD.

He also hopes that sustained delivery through gene therapy can match the outcomes seen over 7 years with the Port Delivery System with ranibizumab (Susvimo; Genentech), data he presented at last year's EURETINA congress.

Khanani said he looks forward to the data from all 3 programmes over the coming 12 months