Descripción del proyecto
Over 30 million Europeans are blind or visually impaired, leading to reduced quality of life and a tremendous loss of productivity in society. Corneal blindness is the second largest cause of blindness globally and while treatable, millions remain unnecessarily blind due to issues of access to transplantable tissue, lack of standardized treatments, and the lag in translating new regenerative medicine therapies to the clinic. The objective of ARREST BLINDNESS is therefore to develop and validate new regenerative-based therapies addressing a spectrum of blinding disorders of the cornea. These conditions either have no effective current treatments, depend on a scarce supply of donor tissue, or non-standardized methods are hindering validation of promising regenerative treatments. To achieve our objective, we will implant GMP-fabricated collagen-based bioengineered scaffolds to replace or regenerate the corneal stroma in cases of stromal thinning, scarring, dystrophy or trauma; deliver therapeutic epithelial stem and endothelial cells to the cornea to restore its transparency; deliver regenerative factors to promote neural growth and function; and actively maintain corneal immune privilege in high-risk situations by targeted therapeutic approaches to regress blood and lymphatic vessels. We will additionally develop advanced methods to image and monitor therapy throughout the cycle from GMP-compliant cell and scaffold preparation through the pre- and intra-operative stages, to postoperative follow-up and evaluation. After proof-of-concept and preclinical validation of key enabling components, these technologies will be used by one or several partners in preclinical models and in phase I/II human clinical studies. ARREST BLINDNESS directly addresses the translation of regenerative medicine, bio-artificial organs, tissue engineered scaffolds, and advanced cell and gene therapies into clinical use and will help to alleviate the worldwide problem of corneal blindness.