mRNA-based gene therapy for retinal diseases

Gene therapy is an exciting therapeutic option which allows for the possible cure of an increasing number of genetic diseases and the delay of the progression of several chronic diseases. Ophthalmology has been a pioneering field in developing these new therapies with one already FDA-approved treatment. However, high cost, immunogenic response, and long-term toxicity with viral vectors remain a concern. Moreover, there is an acute need to develop affordable novel methods to achieve similar results and to allow a more significant number of patients to access these treatments, particularly in Low- and Middle-Income Countries. We are adapting the in vitro transcribed (IVT) mRNA technology to ocular gene therapy mediated by non-viral vectors. The combination of these two technologies will pave the way for the development of specific low-cost gene-based treatments for ocular diseases, from gene replacement and gene knock-out in hereditary retinopathies to the retardation of age-related vision diseases and other common conditions such as glaucoma and dry-eye disease.