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TargetAMD – New treatment approach

Age-related macular degeneration (AMD) is a neurodegenerative disease of the retina, which occurs in two distinct forms, the slower progressive non-exudative form and the rapid progressive exudative form. Merely 20% of the AMD cases belong to the exudative form; however, it is responsible for 90% of cases of severe visual loss.  The prevalence of AMD rises with age. Already in the group of people aged of 60 years 5-10% present early stages of AMD, in those aged 85 years or older about 50% show signs of the devastating disease, determining ageing as the main risk factor for blindness in industrialised countries.

Exudative AMD is characterized by choroidal neovascularisation (CNV) promoted by a protein imbalance. However, due to the short half-life of proteins, current protein-based therapy needs to be repeated monthly. Besides, many patients do not receive adequate treatment, since approved drugs are expensive and not always reimbursed. In addition to the high costs, the life-long intravitreal injections are accompanied by a number of further burdens on the patient from the logistics of the frequent visits at the clinic to negative side effects and acquired tolerances. Therefore, an alternative regenerative cell therapeutic approach is required to preserve patient’s vision. Psychological health and functional status of the individual will be affected in a positive way restoring patient’s ability to carry out most of his/her usual every day activities and to stay mobile.

Academic scientists, industry and SME partners will promote personalised medicine to produce innovative gene delivery technologies, reagents and devices to be translated into one simple and safe gene therapeutic treatment for exudative AMD, funded within the 7th Framework Programme of the EU. The regenerative gene therapeutic approach of TargetAMD is based on the enhanced Sleeping Beauty transposon system (SB100X) which is safe, feasible and efficient. The First-In-Man trial using the SB100X vector in Europe will be promoted and the competitiveness of European science enhanced by the development of Advanced Medicinal Therapy Products (ATMP) using state-of-the-art transposon-based, non-viral transgene delivery technologies. The TargetAMD consortium will communicate the regulatory work and ethical issues of this ATMP on European and International level as well as to patient organisations.

From a social and health systems’ perspective, the new therapeutic approach of TargetAMD will decrease public expenses and increase the overall number of treated AMD patients, especially in EU countries with limited access to ophthalmologists and/or capacity problems.

The gene therapy concept of TargetAMD develops as well innovative instruments, devices and reagents which can be used by any other ophthalmic clinical centres. Moreover, the TargetAMD technology could be adapted to other diseases, for research efforts or gene therapeutic trials.

The novel treatment for AMD, developed by the TargetAMD consortium, is based on the SB transposon system and provides ground-breaking advances in ophthalmic practice and has, based on the newly generated devices and reagents, positive effects on new business opportunities for SMEs and generates jobs and growth in the EU.