Supplementary file 4_Proceedings of the second annual meeting of GenE-HumDi (COST Action 21113).docx

Genome editing for the treatment of human disease (GenE-HumDi) is an EU-funded COST Action for the development and consolidation 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 the discovery, validation, optimization, 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.

人类疾病治疗基因组编辑（GenE-HumDi）是一项由欧盟资助的欧洲科学与技术合作（COST）行动计划，旨在搭建并完善学术、工业与医疗健康领域的反馈网络，以加速、推动并协调基因组编辑（GE）疗法的审批流程。该项目提供学术交流资助金，支持教育培训课程，并举办各类会议与研讨会，以促进活跃于基因组药物研发、验证、优化、生产与临床应用领域的合作方之间及跨领域的协同互动。此外，GenE-HumDi还为青年及早期职业科研人员提供兼具支撑性与国际一流水准的科研环境，以推动基因组编辑领域内的学术网络构建与国际合作。本次数据集汇编了2024年于塞浦路斯利马索尔举办的第二届GenE-HumDi年度会议的会议论文集。为期三日的会议中，该领域知名专家为70余名GenE-HumDi正式成员及非成员科研人员带来了领域最新研究成果与在研项目的前沿进展汇报，围绕领域发展现状展开研讨，并明确了GenE-HumDi行动计划的优先事项，以推进基因组编辑药物的研发进程。七个专题研讨小组梳理了基因组编辑新工具、递送方法、安全监测、临床应用验证、工业生产进展及监管议题等方向存在的知识缺口、标准化短板与传播不足。与此同时，本次会议的宣传推广也助力了基因组编辑技术在人类疾病治疗领域的科普与应用传播。因此，本文所总结的本次会议结论，可作为通过协同协作、强化合作并聚焦关键研发进展，推动基因组编辑技术在欧洲落地应用的行动指南。