Periodic Reporting for period 1 - iPhotoreceptors (Inducible Human Photoreceptors for Research, Development and Therapy to Prevent Blindness)
Période du rapport: 2021-04-01 au 2022-09-30
One of the major causes of adult visual impairment and blindness in industrialized countries is the progressive dysfunction and death of photoreceptor cells 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 have been suboptimal for translating results from the bench to the bedside. The lack of human photoreceptors in sufficient quantity and quality impedes drug discovery to overcome human photoreceptor degeneration. To this end, my team and I have developed within the ERC starting grant ‘ProNeurons’ a disruptive technology to differentiate human induced pluripotent stem cells to photoreceptor precursor cells. This is done by overexpressing three transcription factors yielding in up to 60% in only 10 days. Within the PoC ‘iPhotoreceptor’ actions, we have striven for bringing the induced photoreceptors closer to the market. We have finalized key product development steps and freedom-to-operate reviews to define our final products such as cryopreserved photoreceptors for research and development and therapy. We have identified and approached industrial and academic exploitation partners for buying our products. Our patent application is currently pending. In summary, our technology facilitates fast, efficient and unlimited production of photoreceptors to be used as essential testbeds for drug screenings. This technology has a clear market value and its exploitation will be continued.