FDA Approves Neurotech’s Cell Therapy Rare Eye Disease

The U.S. Food and Drug Administration approved Neurotech Pharmaceuticals cell therapy for the treatment of the degenerative retinal disease macular telangiectasia type 2.

Macular telangiectasia type 2 (MacTel), or idiopathic macular telangiectasia type 2, is a bilateral, neurodegenerative disease in adults with characteristic localized retinal degeneration. It causes the gradual loss of cells in the retina, resulting in vision loss and secondary alterations of the retinal vasculature, the network of blood vessels that supplies oxygen and nutrients to the retina.

Neurotech’s ECT platform is a cell-based gene therapy delivery system designed to provide long-term, sustained delivery of therapeutic proteins to treat chronic eye diseases. The platform consists of a small, semi-permeable capsule containing proprietary allogeneic retinal pigment epithelium cells genetically engineered to produce specific therapeutic proteins for targeted disease treatment. The capsule is surgically implanted. Once in place, the capsule’s semi-permeable exterior membrane allows essential nutrients to enter while also permitting the therapeutic proteins to exit into the eye, where they can travel to the retina located at the back of the eye. The exterior membrane protects the encapsulated RPE cells from the host’s immune system, contributing to their survival and functionality over time.

Encelto is expected to be available in the United States for patients starting in June of 2025.

The approval was based on results from two phase 3 trials that demonstrated that after placement of the Encelto implant significantly slowed the loss of macular photoreceptors in MacTel patients over 24 months.

“This is a historic moment for the MacTel community, as Encelto becomes the first-ever FDA- approved treatment for this vision-threatening disease,” said Thomas Aaberg Jr, chief medical officer of Neurotech. “For those who have been affected by MacTel and for all who have supported this journey, today we look forward to a future where vision loss from MacTel may be slowed.”