Proceedings of the second annual meeting of GenE-HumDi (COST Action 21113)

Abstract

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.

Keywords: ATMP regulation; gene therapy; genome editing; harmonization; molecular medicine; scientific network; standards; translational medicine.

FIGURE 1

Genome Editing for Treatment of Human Disease Network structure. COST Action 21113 is organized in a way that builds on the aim of coordinating and harmonizing procedures and of promoting new findings and identifying constraints covering all stages of GE medicine development from conception to clinical application, as shown for working groups (WG) 1 through 7. The workload of the network is split across these WGs, with each corresponding to overlapping stages in the process of bringing GE-based ATMPs to patients. Besides, to improve the outreach to and inclusion of specific population targets; ITC, Early Career, Grant Award, and Science Communication coordinators are appointed to promote their specific tasks across all WGs.

FIGURE 2

Cost Action and second GenE-HumDi annual meeting demographics. (A) Cost Action Members from NCC, ITC, and non-ITC EU in the GenE-HumDi Action and (B) corresponding attendance at the conference and meeting, where ITC/NCC reached 30% of young members and approximately 18% of members over 40 years old. (C) COST Action 21113 balanced gender representation and (D) corresponding gender distribution for the second Annual Meeting, with gender parity for young researchers and approximately 31% women among participants 40 years of age or older.