Retinal degenerative diseases share the common feature of photoreceptor cell damage, which leads to poor visual function and a gradual loss of these light-sensing cells. This group of degenerative diseases include age-related macular degeneration, retinitis pigmentosa, Stargardt disease, and several others. Human pluripotent stem cell (iPSC)-derived photoreceptor cells may be used to study the etiopathology of these diseases and be used for drug discovery and drug testing. The cells can also be transplanted into animal models to evaluate cell replacement therapies and cGMP manufactured cells may be used in clinical trials
XCell’s fully characterized early stage retinal progenitor cells (RPC) (XR-RPC-001-1V), are derived from a cGMP iPSC line NCL2. They are manufactured using our cGMP-compatible process that has a common intermediate for retinal pigment epithelium (RPE) and RPC. These early stage RPC can be expanded and efficiently differentiated into light-sensing photoreceptors (both rods and cones) using XCell’s retinal differentiation medium (XR-RD-002-PACK).
Our cells have been successfully used in in vitro assays and importantly, following subretinal transplantation into immunodeficient host mouse eyes, our RPC successfully integrated into the photoreceptor layer of the retina and further differentiated into mature photoreceptors. Their fate could be easily followed using the integrated GFP construct.