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A one-and-done injection for wet AMD? Phase 2 results for SAR402663, an investigational gene therapy, show a 97% reduction in treatment burden with a single intravitreal shot. More than 90% of patients on the medium dose needed zero supplemental anti-VEGF injections, and the safety profile looked solid — no serious ocular adverse events reported.
A single injection for wet AMD? That's the promise of SAR402663, Sanofi's investigational AAV2 gene therapy, which delivered impressive phase 2 interim results at the Euretina congress in Vienna. The therapy works by secreting sFLT01, a VEGF-neutralizing protein that curbs abnormal blood vessel growth — all from a single in-office intravitreal injection.
The medium dose stood out: patients saw a 97% reduction in treatment burden, with more than 90% requiring no supplemental anti-VEGF injections at all. Functional and anatomic outcomes were also positive, and the therapy was well tolerated — no cases of endophthalmitis, vasculitis, hypotony, or choroidal effusions were reported. A 19-week corticosteroid prophylaxis was used to manage gene therapy's known immune risks, and the few intraocular inflammation events that did occur resolved with treatment.
By the Numbers:
90% of medium-dose patients needed no supplemental anti-VEGF injections
Why it matters: Wet AMD patients currently face a demanding cycle of frequent anti-VEGF injections — sometimes monthly. A single gene therapy injection that could replace years of repeat treatments would be a meaningful advance for patients and clinicians alike. Longer follow-up data at 52 weeks and beyond will be critical to confirming durability.
