Development of a novel therapeutic treatment to engineer corneas to resist graft rejection

Corneal transplantation is one of the most successful transplant procedures, due mainly to the relatively immune-privileged status of the eye and the fact that the cornea is largely free of blood vessels. However there is a failure rate in the first year of 14% and much higher in high rejection risk patients (such as those with failed previous grafts) due to blood vessel fromation in the patients eye, and the replacment of failed grafts is the indication for corneal transplantation for a significant proportion of patients in several referral centres. The prognosis in these patients can be so poor that many are not offered the opportunity of a further transplant and are left blind. EncorStat® is a novel engineered donor cornea, modified prior to transplantation, to extend rejection-free survival or prevent this risk entirely by suppressing blood vessel formation into the cornea post-transplant. We propose to use TSB funding to complete non-clinical safety studies and gain regulatory approval for clinical evaluation, to produce clinical grade vector and to use this to evaluate EncorStat® in a Phase I/IIa clinical trial.