Генотерапия 4D-150 сократила нагрузку от лечения влажной возрастной макулярной дегенерации на 78%

4D-150 gene therapy reduces treatment burden in wet AMD Key takeaways: - There was a 78% reduction in treatment burden compared with aflibercept every 8 weeks. - Nearly half of eyes went 2 years without an additional aflibercept injection. MONTREAL — In a phase 2b study, an intravitreal gene therapy reduced the treatment burden in patients with neovascular age-related macular degeneration, according to a study. “4D-150 is an [adeno-associated virus] that has evolved to cross retinal barriers well, to have high gene expression and to minimize immune reaction,” Carl C. Awh, MD, FASRS, said in a presentation at the American Society of Retina Specialists annual meeting. “It delivers a dual transgene payload resulting in production of aflibercept and an inhibitory RNA against VEGF-C.” Patients received one 2 mg intravitreal aflibercept injection 7 days before the gene therapy. One 4D-150 injection (4D Molecular Therapeutics) was administered on day 1 followed by 2 mg of aflibercept on day 28. There were two 4D-150 groups: 15 patients who received 1E10 vg per eye and 30 who received 3E10 vg per eye, according to the study abstract. Researchers compared these regimens to aflibercept given every 8 weeks, Awh said. Both doses of 4D-150 demonstrated good maintenance of vision and anatomy with slightly better results in the higher-dose group. Awh said the 3E10 vg per eye dose is the subject of ongoing phase 3 trials. Among patients in the 3E10 vg per eye group, researchers found a 78% reduction in treatment burden compared with aflibercept every 8 weeks. Additionally, 46% of eyes went 2 years without an additional aflibercept injection, and 69% had no more than two aflibercept injections over the course of 2 years after receiving 4D-150, Awh said. In a subgroup of 15 patients who had a mean time since neovascular AMD diagnosis of 2.4 months, 66% of eyes went 2 years without an additional aflibercept injection, and 87% had two or fewer aflibercept injections after a single injection of 4D-150, according to the study. Awh said the therapy was safe, with just two eyes experiencing inflammation during the first 28 weeks of the study. There were no other cases of severe adverse events through 4 years. “This combination of safety and this tremendous reduction in patient burden is exciting,” Awh told Healio. “I look forward to phase 3 outcomes, which should be reported sometime in 2027.” For more information: Carl C. Awh, MD, FASRS, is a retina specialist with Tennessee Retina in Nashville, Tennessee.