After she received her M.D. and Ph.D. from Kyoto University, served as an assistant professor in the Kyoto University Hospital. Discovered the potential of stem cells as a tool for retina therapy when she moved to the Salk Institute, U.S. She joined RIKEN in 2006, her team launched a pilot clinical study using iPS cells first in human in 2013. She started a new carrier, a president of start up company; Vision Care Inc., to proceed implement clinical therapy.
The World’s First Allogeneic iPS-derived Retina Cell Transplant
Our aim is to develop outer retinal cell therapy using iPS cells. The first in man application of autologous iPS cell-derived RPE (iPSC-RPE) cells started in 2013, targeted age-related macular degeneration (AMD). We proceeded to clinical research using HLA matched allogeneic iPSC-derived RPE cells. Immune responses to transplanted allogeneic cells could be suppressed by topical steroid administration without systemic immunosuppressant. Safety was confirmed one year after transplantation of HLA 6 loci matched allogeneic iPSC-RPE transplantation. So far 6 patients have received autologous and allogeneic iPS-derived retinal pigment epithelial cell transplantation. The grafted cells survive without any harm in all the patients.
The next challenge is photoreceptor replacement. We proved that grafted photoreceptor cells formed synapses only when they were transplanted in the form of organoids. They showed the functional recovery in the completely photoreceptor degenerated blind mice after transplantation. With those findings as POC, we are performing clinical study using retinal organoid for retinitis pigmentosa.