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Gene Therapy: From Birth to Maturity Requires Commitment to Science and Ethics.

OVER THE LAST 25 YEARS, gene therapy has become a reality, since inherited disorders of the hematopoietic system, notably primary immunodeficiencies, hemophilia B, and inherited disorders of the retina have been corrected by ex vivo or in vivo gene transfer with a sustainable clinical benefit. Development of chimeric antigen receptor T cells as a successful strategy to treat B cell malignancies adds a new dimension to gene therapy, with bright prospects. It is perhaps worth analyzing what the factors of success have been. Obviously, many contributions have made these advances possible, starting with the work of the pioneers in molecular biology, then virology, stem cell biology, and refined delineation of the pathophysiology of targeted diseases. This should not be forgotten at a time when genome editing may open new options. Gene and cell therapy can only be based on very solid science and integrate medical needs. It is necessary to build multi-competent teams, a key for innovation and success. The field also needs to assess the achievements carefully, with rigorous rules in terms of communication also being required in order to avoid conflicts of interests in a setting of growing academic/industry partnerships. There is also a necessity to work to make the coming products of gene therapy accessible to everyone who needs them, a serious challenge for the years to come. Finally, ethical aspects particularly regarding the potential development of germline genome editing, as advocated by some, need to be discussed in depth within the gene therapy community but also far beyond. Cell and gene therapy carry a bright scientific perspective that must be accompanied by a careful ethical review process of its development for the benefit of patients. Given the contribution by European scientists to the field, the European Society of Gene and Cell Therapy has obviously been and should continue to be at the forefront of both science and reflection upon its applications to medicine.