Proceedings From Second Annual GenE-HumDi (COST Action 21113) Meeting

María Ortíz-Bueno¹Iris Ramos-Hernández¹Luis Algeciras Jiménez¹Nechama Kalter²Juan Roberto Rodriguez-Madoz^3,4,5José Bonafont⁶Rajeevkumar Raveendran Nair⁷Oliver Feeney⁸Laura Torella⁹Lluis Montoliu^10,11Petros Patsali¹²Claudio Mussolino^13,14Yonglun Luo^15,16Alessia Cavazza^17,18Merita Xhetani¹⁹Ayal Hendel^2*Karim Benabdellah^1*Carsten Werner Lederer^12*Francisco Javier Molina-Estevez^20,21,22*

• ¹Centro Pfizer - Universidad de Granada - Junta de Andalucia de Genomica e Investigacion Oncologica, Granada, Spain
• ²Institute of Nanotechnology and Advanced Materials, Bar Ilan University The Mina and Everard Goodman Faculty of Life Sciences, Ramat Gan, Israel
• ³Clinica Universidad de Navarra Cancer Center, Pamplona, Spain
• ⁴Haemato-Oncology Program, Clinica Universidad de Navarra Centro de Investigacion Medica Aplicada, Pamplona, Spain
• ⁵IdiSNA, Universidad de Navarra, Pamplona, Spain
• ⁶Danaus GT, Madrid, Spain
• ⁷Norges teknisk-naturvitenskapelige universitet Kavli Institute for Systems Neuroscience, Trondheim, Norway
• ⁸Research Unit "Ethics of Genome Editing", Institute of Ethics and History of Medicine, Universitat Tubingen Institut fur Ethik und Geschichte der Medizin, Tübingen, Germany
• ⁹DNA & RNA Medicine Division, Clinica Universidad de Navarra Centro de Investigacion Medica Aplicada, Pamplona, Spain
• ¹⁰Centro Nacional de Biotecnologia Departamento de Biologia Molecular y Celular, Madrid, Spain
• ¹¹Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid, Spain
• ¹²Molecular Genetics of Thalassemia Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus
• ¹³Institute for Transfusion Medicine and Gene Therapy, Universitatsklinikum Freiburg, Freiburg, Germany
• ¹⁴Center for Chronic Immunodeficiency (CCI), Universitatsklinikum Freiburg Institut fur Immundefizienz, Freiburg, Germany
• ¹⁵Department of Biomedicine, Aarhus Universitet, Aarhus, Denmark
• ¹⁶Steno Diabetes Center Aarhus, Aarhus Universitetshospital, Aarhus, Denmark
• ¹⁷Department of Medical and Surgical Sciences for Children and Adults, Universita degli Studi di Modena e Reggio Emilia, Modena, Italy
• ¹⁸NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom
• ¹⁹Department of Biology, Faculty of Natural Science, Universiteti i Tiranes, Tirana, Albania
• ²⁰Instituto de Investigacion Biosanitaria de Granada, Granada, Spain
• ²¹Universidad de Granada Facultad de Farmacia, Granada, Spain
• ²²Universidad de Granada Departamento de Biología Celular, Granada, Spain

Abstract Genome editing for the treatment of human disease (GenE-HumDi) is an EU-funded COST Action for reinforcement of academic, industrial and healthcare feedback networks aiming to accelerate, foster and harmonize the approval of genome-editing (GE) therapies. GenE-HumDi offers mobility grants, supports educational courses, and hosts conferences and meetings to promote synergistic interactions among and across partners active in discovery, validation, escalation, manufacturing and clinical application of genomic medicines. Furthermore, it provides young and early career scientists with a supportive and world-class environment to foster networking and international collaborations within the GE field. We compiled the proceedings of the second Annual GenE-HumDi Meeting held in Limassol, Cyprus, in 2024. Over three days, renowned experts from the field updated an audience of over 70 GenE-HumDi members and non-member scientists on the latest discoveries and ongoing projects, discussed the status of the field, and identified GenE-HumDi action priorities to advance research and development for GE medicines. Seven focused discussion groups identified gaps in knowledge, standardization and dissemination for new GE tools, delivery methods, safety monitoring, validation for clinical use, and progress in industrial manufacturing and regulatory issues. Simultaneously, publicity about the event itself contributed to outreach and dissemination of GE for human diseases. Therefore, the conclusions of that meeting, summarized here, serve as a compass toward GE application in Europe through coordination, enhanced collaboration and focus on critical developments.