Résumé
Gene therapy represents the latest innovation among advanced therapy medicinal products. The first gene therapies were launched in Europe in the early 2010s to treat serious disorders such as monogenic diseases. There are several challenges surrounding their development, production and commercialisation. The example of X-linked juvenile retinoschisis is used here to understand these challenges. The field of gene therapy is still in its early stages. Even though several drugs are already on the market, many unknowns remain. Firstly, most of these medicines were granted marketing authorisation on the basis of noncomparative clinical trials involving few patients, making it difficult to assess their efficacy and safety. One of the main unknowns concerns the durability of the effect of gene therapies. Secondly, research into the production processes for gene transfer vectors is continuing, as a high need for standardisation and cost rationalization remains. Lastly, very high initial investment and production costs have led manufacturers to negotiate high prices, in the million-euro range, to ensure the economic viability of these therapies, which goes against the current political and economic context, which aims to reduce healthcare spending. In conclusion, gene therapies face many challenges at every stage of their development, raising the question of their availability to patients, as well as their potential future democratisation.