Moorfields Stem Cell Treatment Improves Vision for Aniridia-Related Keratopathy
A novel cellular therapy trial conducted at Moorfields Eye Hospital NHS Foundation Trust in London is evaluating an artificial tissue known as RAFT-OS (Real Architecture for 3D Tissues Ocular Surface) for patients with aniridia-related keratopathy, a complication of a genetic eye condition present from birth.
- The clinical trial investigates RAFT-OS, an engineered cellular tissue populated with limbal epithelial and stromal cells, designed to treat aniridia-related keratopathy (ARK) in 21 patients.
- Adult limbal and stromal donor cells are sourced from NHS Blood and Transplant, Tissue and Eye Services in Liverpool, before being manufactured by the Cells for Sight Stem Cell Therapy Research Unit at the UCL Institute of Ophthalmology.
- Trial participants undergo 10-weeks of immune suppression therapy prior to transplantation and are followed up to 12 months after transplantation.
Clinical Background and Pathogenesis of Aniridia-Related Keratopathy
Aniridia is a genetic eye condition present from birth that can lead to aniridia-related keratopathy (ARK). To address this, researchers designed the RAFT-OS therapy to rebuild the ocular surface using bio-engineered tissue constructs.
Study Design and Administration of the RAFT-OS Cellular Therapy
According to clinical trial records from the Veeva Clinical Trials database, the trial operates as a seamless Phase I/II single-dose, single-arm study evaluating if RAFT-OS is a safe and effective alternative treatment in 21 patients. The therapy is manufactured by the Cells for Sight Stem Cell Therapy Research Unit at the UCL Institute of Ophthalmology. Adult limbal and stromal cells utilized in the artificial tissue are supplied by NHS Blood and Transplant, Tissue and Eye Services located in Liverpool. Following screening, participants commence 10-weeks of immune suppression therapy to prepare for the transplantation. The RAFT-OS construct is then surgically transplanted into the patient’s worst-affected eye at Moorfields Eye Hospital NHS Foundation Trust.
Safety Monitoring and Post-Operative Patient Triage
Rigorous patient monitoring follows the surgical intervention. Trial protocols mandate safety assessments at days 1, 7, 14, 21, and at the one-month mark to track intermediate or major safety events. Participants continue immune suppression therapy and remain under clinical observation for up to 12 months after transplantation. Managing complex corneal degenerations requires specialized ophthalmic oversight and structured multi-disciplinary care pathways.
Future Outlook for Ocular Regenerative Medicine
*Disclaimer: The information provided in this article is for educational and scientific communication purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider regarding any medical condition, diagnosis, or treatment plan.*