OPTICELL

In developed countries, the main causes of blindness and visual impairment are retinal diseases, including Age-related Macular Degeneration (AMD) and Retinitis Pigmentosa (RP). These diseases, which lead to a progressive loss of photoreceptors—the light-sensing cells of the retina—affect more than 30 million people worldwide. Some existing treatments aim to slow the progression of certain forms of these diseases but do not restore vision once photoreceptors are lost. Therefore, developing new therapeutic strategies is essential.
Cell or tissue therapy based on stem cells offers promising prospects for replacing retinal pigment epithelium (RPE) cells or/and photoreceptors lost in these pathologies. Therapies using RPE cells derived from human pluripotent stem cells (hPSCs) have been tested in phase I/II clinical trials, but it remains crucial to increase the production of these cells for future industrial production. In cases where photoreceptors are also lost, their replacement is necessary to restore the retina’s photosensitivity. Photoreceptors can be produced from hPSCs via 3D retinal organoids, but their functional maturation after transplantation remains a challenge. The differentiation of hPSCs into retinal cells remains a long and complex process, requiring meticulous manipulations and numerous quality controls that are often destructive.
The OptiCELL project, which brings together and combines multidisciplinary expertise (retinal cell and tissue therapy, stem cell bioproduction, polymer biotechnology, and AI applied to living systems), aims to address these various challenges. The partners in this project have recently developed alternative retinal outer layer cell therapy products derived from hPSCs that integrate optogenetics and tissue engineering, enabling the transplantation of functional cells.
To advance to the preclinical and clinical phases, our objectives are to:
– Scale up production to comply with Good Manufacturing Practices (GMP)
– Improve final products targeting photoreceptors and/or RPE.
– Develop non-destructive and low-cost quality controls via AI.
The development of all these steps will pave the way for future industrial production, compatible with the large number of patients awaiting treatment, and will represent a major advance in the field of Advanced Therapy Medicinal Products.