Breakthrough Gene Therapy Implant Encelto Approved for Rare Eye Disease

The U.S. Food and Drug Administration (FDA) has approved Encelto, a groundbreaking gene therapy implant designed to combat macular telangiectasia type 2 (MacTel), a rare eye disease that leads to vision loss. Developed by Neurotech Pharmaceuticals, Encelto represents the first and only implantable therapy utilizing cell technology to deliver a crucial protein that helps slow the disease's progression. The approval, granted on March 7, 2025, marks a significant milestone in the treatment of MacTel, offering new hope to those affected by this degenerative condition.

Encelto is a diminutive implant containing lab-grown retinal cells engineered to release the protein revakinagene taroretcel. This protein plays a vital role in decelerating the deterioration of light-sensing macular cells in MacTel patients. The macula, an essential component of the eye responsible for sharp central vision, is critical for performing everyday tasks such as reading, writing, driving, and recognizing faces. Over two years, Encelto demonstrated its efficacy in slowing macular cell loss in two phase III clinical trials, providing a promising new treatment avenue for individuals grappling with this progressive disease.

Despite its potential benefits, Encelto is not a cure for MacTel but rather a treatment option aimed at halting the disease's advancement. Patients considering Encelto should consult their doctors about any existing eye infections before proceeding with the treatment. The implant is expected to be available to patients in the United States by June 2025, expanding the arsenal of therapies available to combat vision impairment caused by MacTel.

MacTel is a rare eye disorder that gradually damages the macula, leading to vision deterioration over time. By targeting the disease's underlying mechanisms, Encelto offers a novel approach to managing this challenging condition. The introduction of Encelto underscores the potential of innovative gene therapies to transform treatment paradigms and improve patient outcomes.