FDA Approves Encapsulated Cell Therapy for Degenerative Eye Disease

I’m excited to announce the approval of Encelto, a first-of-its-kind implantable cell therapy that is also the first and only approved treatment for adults with the devastating neurodegenerative retinal disease, macular telangiectasia type 2 (MacTel).

MacTel is a progressive disease that occurs when blood vessels around the center of the eye’s macula become abnormal, leading to vision loss in adults.

While many cell and gene therapies provide a “one and done” pathway to treatment, Encelto (revakinagene taroretcel-lwey) is designed to continuously deliver therapeutic proteins to slow MacTel’s progression. This innovative approach also opens the door to future treatment possibilities for other rare diseases.

The treatment works by using a cell-based system to deliver ciliary neurotrophic factor (CNTF) locally in the retina. A semi-permeable capsule is surgically implanted into the vitreous cavity of the eye. The capsule contains allogeneic retinal pigment epithelium (RPE) cells genetically engineered to continuously produce CNTF in the retina. CNTF promotes the survival and maintenance of photoreceptors, which slows degeneration and can potentially improve long-term vision for patients.

The developer of Encelto, Neurotech Pharmaceuticals, Inc., expects the therapy to be available to U.S. patients starting in June.

Data from two phase 3 studies on Encelto (NT-501) showed that the implant was durable and well tolerated, with a manageable safety profile. After the implant placement, Encelto significantly slowed the loss of photoreceptors in patients after two years.

I want to congratulate everyone involved in the research and development of Encelto and I’m thrilled for the patient community who will be able to benefit from this unique treatment in the coming months!

Dr. Cannon is ASGCT President.

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