FDA Approves First Gene Therapy Eye Implant

In March 2025, the U.S. Food and Drug Administration approved Encelto (revakinagene taroretcel), the first-ever gene therapy implant for patients with macular telangiectasia type 2 (MacTel Type 2). This milestone marks a pivotal advance in treating a rare and progressive retinal disease that damages photoreceptors, leading to central vision loss.

Encelto is an allogeneic encapsulated cell-based implant inserted directly into the eye during a single surgical procedure. It works by continuously releasing ciliary neurotrophic factor (CNTF)—a protein with neuroprotective properties—directly to the macula, supporting cell survival and slowing degeneration.

Why This Matters

• Breakthrough for Rare Eye Disease
  MacTel Type 2 has long lacked effective treatments. This implant offers a first-of-its-kind therapeutic option for patients facing progressive vision loss.

• Long-Term Protection With a Single Procedure
  Unlike repetitive injections, Encelto delivers sustained therapy within a single implant, improving compliance and quality of life.

• Expanded Gene Therapy Landscape
  This approval signals growing confidence in gene-driven treatments and encourages future innovation in ocular and other degenerative diseases.