It could arrive as early as 2027, but there is the problem of costs
There wet maculopathy It is an age-related degenerative disease of the retina capable of rapidly compromising vision, with a significant impact on the quality of life. It hits millions of people in the world and represents one of the main causes of vision loss in the elderly population. Today, available treatments include periodic injections of anti-VEGF drugs, which however require an average of 5–7 administrations per year. But it could arrive soon a long-term curethanks to an innovative gene therapy.
A single injection for maculopathy
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The results of preliminary studies and more advanced trials are largely positive. After the first administration with the new therapy, in many patients no further injections were required in the four years of follow-up. The credit goes to 4D-150 gene therapies And ABBV-RGX-314presented at the FLORetina Icoor congress which brought together 6000 experts from 70 countries in Florence. The goal is to get approval from the Food and Drug Administration by 2027.
gene therapy for maculopathy
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Gene therapy involves the introduction directly into the cells of the retina of therapeutic DNA, i.e. equipped with the instructions to produce the cells autonomously anti-Vegf proteins. These are molecules that counteract the growth factors responsible for the proliferation of abnormal blood vessels that damage vision. Today anti-VEGF drugs must be injected from the outside several times a year; with this technology, however, production would be continuous and automatic.
Updated studies on ABBV-RGX-314 and 4D-150 show lasting results in front of a robust security profile.
costs and availability
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Phase III studies, called Atmosphere And Ascentare still underway in the United States and in several European countries, including Italy, which also has a high number of participants. In our country there has been experimentation started on 12 patientsto which another 4 will be added, for a total of 1,200 people in more than 200 centers. Preliminary data is expected in the fourth quarter of 2026 but remains to be resolve the issue of reimbursement: gene therapies in fact have high costs capable of putting healthcare budgets under pressure.
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