CA21113 - Genome Editing to Treat Humans Diseases (GenE-Humdi)

Recent advances on genome editing (GE) technologies have opened the possibility of treating diseases through precise modifications of patients’ genomes. Impressive results have been achieved on animal models of several genetic disorders, infectious diseases as well as cancer and several clinical trials are already on going. However, the inadequate integration of the results of academic research into the research development strategy of pharmaceutical companies, the insufficient interest of academic institution in regulatory science and the absence of established standards to well acceptable risk-benefit ratio by regulatory agencies, preclude its general application for treating human diseases. Therefore, the translation of the GE technologies to address public health needs, require a strong collaboration between basic and clinical research, regulatory bodies and the different stake holders involved for each application. There are several networks to improve or analyse GE technologies for different applications, however, no one cover all the actors involved in gene therapy translation. The principal aim of the GenE-HumDi Action is to bring together pharmaceutical companies, academic institution, science and regulatory agencies, biotechnology firms, patient advocacy association and information technology, in order to tackle knowledge fragmentation with the aim to accelerate the translation of GE technologies to the treatment of human diseases.