Medical need and cell-based therapeutic approach

AMD is the most common cause of blindness in developed nations and affects around 200 million people worldwide. As the disease progresses, the cone photoreceptor cells of the retina – which are crucial for central and colour vision – are lost in particular.

Existing treatments can only slow the progression of the disease; they cannot reverse it. Against this backdrop, ReRetina2 pursues a regenerative approach based on human induced pluripotent stem cells (hiPS cells). These are used to produce cone photoreceptor cells. A newly developed process enables efficient production within around three weeks.

Preclinical validation and prospects for application

During the initial feasibility phase of the GO-Bio project, the functionality of the generated cells is to be investigated in a preclinical AMD pig model. The focus is on integration into the retina, functional activity and initial indications of a possible restoration of visual function.

In addition, safety aspects will be assessed and the foundations for scalable production will be established. The aim is to demonstrate the technological feasibility of a cell replacement therapy for AMD and to lay the groundwork for subsequent clinical development.

To learn more about funded GO-Bio projects, visit the overview page.