Genome Editing for the treatment of human Disease Network

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2025 Course: Optimizing Gene Editing with Advanced Omics: Safety, Off-Target Detection, and Cell Phenotyping

11-14 of March 2025GENYO Center, Granada (Spain) Presentation The Genome Editing to Treat Human Diseases (GenE-HumDi) COST Action (CA21113), chaired ...
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Welcome to the Genome Editing for the treatment of human Disease Network (GeneHumdi | COST action CA21113) Website. We are Academia and industry scientist, translational scientist, clinicians, regulatory agents, and industry partners dedicated to the improvement and translation of Gene Editing protocols to treat diseases lacking sound medical care or susceptible of increase safety and control of the therapeutic interventions through the implementation of precision gene medicine.

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 GeneHumdi 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. GeneHumdi is supported by COST (European Cooperation in Science and Technology) which is a funding organisation for research and innovation networks operating in Europe since 1971. In their organigram, GeneHumdi nourishes from the ideas exchange of Management Committee (MC) members from over 24 EU Countries and MC and other members are organized into Working Groups (WG) to distribute GeneHumdi workload and allocate specific question to dedicated experts in each field.