Pro­ject status/Results

Tar­get Late AMD

Tar­get Late AMD star­ted in Novem­ber 2022. The pro­ject will be com­pleted in April 2024 and fo­cus on the es­tab­lish­ment of the pro­duc­tion of the cell product, i.e., reti­nal pig­ment ep­ithe­lial (RPE) cells de­rived from auto­log­ous in­duced pluri­po­tent stem cells (iPSC) and ge­net­ic­ally mod­i­fied (trans­fec­ted) to pro­duce thera­peut­ic factors (PEDF and GM-CSF) aim­ing at re­cov­er­ing a healthy reti­nal en­vir­on­ment. Fol­low-up pro­jects will fo­cus on the iPSC generation, qual­ity con­trol meas­ure­ments and clini­cal-grade man­u­fac­tur­ing.

The main ob­ject­ives of the present pro­ject are the:

  • De­vel­op­ment of a ro­bust pro­tocol for the mat­ur­a­tion of iPSC to RPE cells
  • Es­tab­lish­ment of the ge­net­ic modi­fic­a­tion of iPSC-de­rived RPE cells (trans­fec­tion to over­ex­press the pro­teins PEDF and GM-CSF us­ing the Sleep­ing beauty trans­po­son sys­tem)
  • Ana­lys­is of the ef­fi­ciency and the safety of the cell product, i.e., PEDF-GM-CSF-trans­fec­ted iPSC-de­rived RPE cells

In the first months of the pro­ject we were already able to suc­cess­fully ma­ture iPSC to RPE cells and to start propaga­tion of the cells (fig­ure, one cell is red en­circled).

Cul­ture of pro­lif­er­at­ing iPSC-de­rived RPE cells

Target Late AMD iPSC to RPE cells