Work package 9
Performance of an Individual First-in-Human Clinical Trial
A Phase Ib/IIa clinical trial with 10 patients will be performed in which PEDF-transfected autologous iris pigment epithelial (IPE) cells (UNIGE, Switzerland) will be transplanted subretinally in neovascular AMD patients.
Since the harvesting IPE cells is only minimally invasive, this cell population, quite similar to the degenerated RPE cells, will be transplanted to the subretinal space offer genetic modification to insure for tissue regeneration and “drug delivery” (PEDF secretion). Transplantation of non-genetically modified IPE cells to the subretinal space of AMD patients has been done in over 100 patients and has been shown safe. The novel approach is expected to increase the benefits for patients, health care systems and industry.
The TargetAMD protocol developed for the treatment of patients suffering from exudative AMD will consist of the isolation of autologous IPE cells (clinical trial phase Ib / IIa, Switzerland), by a well-established surgically procedure (iridectomy / retinal biopsy). The isolated cells (5’000 – 10’000 cells) will be transfected ex vivo via an electroporation procedure, genetically modifying the cells by a PEDF-encoding plasmid using the SB100X transposon system. Afterwards, the transfected IPE cells will be subretinally implanted, completing the approximately 60 minutes lasting one-session surgical procedure (Fig. 2).
Milestone 5 represents the completion of the first-in-human clinical trial of TargetAMD.