IOO Genetics

We have started a clinical trial to assess the safety of injecting retinal pigment epithelium (RPE) cells, grown from embryonic stem (ES) cells, into the eyes of patients with Stargardt macular dystrophy. This is the first clinical trial in Europe to use cells derived from ES cells, and follows an ongoing study in the USA where two patients received similar cells without any complications to date.

Beginning with patients that are severely visually impaired and designed to test whether the transplants are safe, this trial uses RPE cells that have been turned into RPE cells in a dish - it is the mature RPE cells that are injected, in the hope that they can repair the damaged tissue safely.

So far four patients have undergone the surgery, which went smoothly, and they will be followed over the next two years to ensure the technique is well-tolerated.

Further studies would be required to determine whether vision can also be improved.

People with Stargardt macular dystrophy may be eligible to participate in this trial and in future studies if they have severe sight loss sue to Stargardt and are otherwise well. For further information, please contact us.