The FDA has registered the first implantable gene therapy, Encelto. It slows the degeneration of the macula and prevents vision loss in type 2 macular telangiectasia.

The U.S. Food and Drug Administration (FDA) has approved the first implantable gene therapy, Encelto (revakinagene taroretcel), for the treatment of type 2 macular telangiectasia. This announcement was made by the development company Neurotech Pharmaceuticals.

This rare degenerative eye disease damages the blood vessels that supply the macula (the central part of the retina responsible for sharp vision). Over time, these vessels dilate, forming twists (telangiectasia), which leads to the leakage of fluid into surrounding tissues. At the same time, the nourishment of photoreceptors (light-sensitive cells in the retina that transmit images to the brain) and Müller cells (which ensure the normal function of photoreceptors and retinal neurons) is disrupted. As a result, patients gradually lose the ability to distinguish letters, recognize faces, and eventually lose their vision altogether.

Encelto is a miniature implant containing lab-grown retinal cells. It is surgically implanted in the patient's eye, where it continuously produces ciliary neurotrophic factor (CNTF). This protein helps slow down the degeneration of the macula and delays the onset of blindness. Gene therapy has slowed the loss of photoreceptors among participants in Phase III of two studies over a two-year observation period.

Americans will be able to benefit from this new treatment starting in June 2025.

 

Source: Neurotech