Abstract
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Since the 1990s, and in the wake of sequencing the human genome, Inserm has become one of the pioneers in gene therapy. During the 2000s, breakthrough treatments were tested for severe combined immunodeficiencies, adrenoleukodystrophy, hemophilia B, and beta-thalassemia. Since then, many other trials have broadened the scope of interventions—retinitis pigmentosa, Parkinson's disease, Friedreich's ataxia, and of course cancers. Over the same period, our laboratories have supported the revolution of adult and induced embryonic stem cells, with the challenge of controlling differentiation and specialization. The ability of stem cells to regenerate deficient tissues opens up countless clinical opportunities. In particular, Inserm's researchers have made remarkable advances in macular degeneration linked to age, type 1 diabetes, or the treatment of heart failure.
Today, Inserm is one of the European and global leaders in terms of publications about these gene and cell therapies. Biotherapies are at the heart of a new field of precision—personalized, targeted, and regenerative biomedicine. This is an unprecedented epistemiological, technological, and medical revolution. It results from understanding the molecular mechanisms of the living being and the ability to correct their pathological trajectory in the body. There are numerous implications for organizing research and treatment. The new biomedicine demands a strong technological component (high-speed screening and sequencing, modeling, multiscale imaging), an ability to work reliably on low demographic cohorts and clinical trials, network integration of the translational chain from the fundamental to the clinical, and an opening to breakthrough innovation in highly regulated fields. In this highly competitive international background, the role of Inserm and its Aviesan partners is to create the conditions for research excellence.
Today biotherapies are one of the frontiers in biomedical innovation. Advances are made possible through certain advantages presented by our research and treatment systems. Here we should mention a structured and very high-quality healthcare system, with varied and clinically well-characterized patient cohorts, effective integration of research in healthcare with a strong translational orientation, networks and structures specialized in treating patients, extensive experience in implementing healthcare plans, an international competitive academic research facility, and high-quality industrial companies and innovative SMEs in our country.
Biotherapies have huge potential: In proportion to their promises they raise social expectations and ethical questions to which Inserm must also respond. For more than 10 years our institute has conducted fundamental work with patient associations, which have become fully fledged players in responsible research mindful of the societal dimensions of its advances. The Inserm Ethical Committee also undertakes pioneering debates on these questions, the breakthrough nature of which overturns our representations and challenges our certainties. The current debate on CRISPR/Cas9 technology is a good illustration.
Since 2001 the French Gene and Cell Therapy Society (SFTCG) has supported the French research community involved in this innovative field. Through its informed discussion with all stakeholders, it plays an essential pivotal role. The quality of the articles compiled in this special edition demonstrates the SFTCG's high level of commitment, and through it the work of French gene and cell therapy researchers.