Project description
Stem-cell derived photoreceptors for research and development in blindness prevention
Retinal degenerative diseases and the death of photoreceptor cells are the major causes of adult visual impairment and blindness. The team of the EU-funded iPhotoreceptors project previously developed an innovative method to differentiate human induced pluripotent stem cells into photoreceptor precursor cells using overexpression of specific transcription factors. This technology allows the fast and unlimited production of photoreceptors essential for research, photoreceptor replacement therapies and drug screenings. The current project aims to finalise the remaining development steps, define the final products, such as photoreceptor differentiation kits and cryopreserved photoreceptors, and work towards the discovery of new drugs for the treatment of retinal degeneration.
Objective
One of the major causes of adult visual impairment and blindness in industrialized countries is the progressive dysfunction and death of photoreceptor cells (PR) as a result of retinal degenerative diseases. Current treatment options are still insufficient to counteract these forms of blindness. The development of many drugs and therapies fail in preclinical stages because the used animal models are suboptimal for translating results from the bench to the bedside. Especially, for testing the transplantation of human PRs, which is currently extensively explored as a treatment option for late stages of retinal degeneration, the biggest bottleneck is the lack of PR material in sufficient quantity and quality. Within the ERC Starting Grant ProNeurons, my team and I have developed a disruptive technology to differentiate human induced pluripotent stem cells (hiPSCs) to PR precursor cells by overexpressing three transcription factors yielding in up to 60% in only 10 days. Our technology allows fast, efficient and unlimited production of PRs essential as testbeds for drug screenings, for further disease research and for photoreceptor replacement therapies. Within the PoC actions, we strive for bringing the induced PRs closer to the market. We will finalize the last product development steps and FTO reviews to define the final products such as PR differentiation kits and/or cryopreserved PRs for research, development and therapy. We will identify and approach industrial exploitation partners for out-licensing or further funding. The final goal of commercializing the induced PRs is to aid the development of new drugs to treat retinal degenerative diseases.
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Programme(s)
Funding Scheme
ERC-POC - Proof of Concept GrantHost institution
53127 Bonn
Germany