During the initial reporting period, the consortium commenced project activities by recruiting staff, installing new equipment, and establishing methodologies and protocols related to Work Packages (WP) 1, 2, and 3. A strong emphasis was placed on communication, dissemination, exploitation activities (WP5), and project management (WP6). The consortium successfully developed an epigenetic editing system delivered via a non-viral strategy. We demonstrated the ability to target formulations more specifically to glial cells and advanced safety assessments in neural cell models, including neurons, microglia, and astrocytes. In vitro safety studies, conducted in accordance with ISO 10993-5:2009(E) guidelines, showed that at tested concentrations, the formulation did not significantly impact cell viability, with over 85% of cells remaining viable after 24 hours. Furthermore, the consortium demonstrated effective gene editing capabilities in cell monolayer cultures. Progress was also made in generating reporter mouse models to facilitate in vivo monitoring of the epigenetic reprogramming potential of the formulations. Toxicity assessments against immune cells in blood are underway. Key findings from these activities have been published in prestigious journals such as Angewandte Chemie, Nature Communications, and Nucleic Acids Research.
To maximize project visibility and impact, a comprehensive communication and dissemination strategy was implemented. This included launching a project website, developing dissemination materials, and publishing three high-impact scientific articles. The consortium participated in eight international scientific meetings, organized a multi-stakeholder workshop, and held bilateral meetings with industry representatives, patient advocacy groups, and healthcare professionals. Additional outreach activities encompassed dissemination via LinkedIn and X (formerly Twitter), engagement at public events (e.g. schools, European Researchers' Night), press releases, and the production of a project explanatory video.
During the first reporting period, the following milestones have been successfully achieved: (i) kick-off meeting held and management structure established; (ii) online platform launched for data sharing and communication among partners; (iii) synthesis of formulations to deliver the epigenetic editing system; (iv) initial results of the in vitro delivery of the epigenetic editing system; (v) initial results related with the in vitro epigenetic reprogramming of brain cells; (vi) generation of a reporter mouse and (vii) organization of a multi-stakeholder workshop.
During this period the following deliverables have been successfully submitted: (i) D1 – Website and project logo; (ii) D2 – Ethics Report; (iii) D3 – Data Management Plan; (iv) D4 – Plan for dissemination and Communication Activities; (v) D5 – Plan for Exploitation Activities; (vi) D6 – Production of the epigenetic editing system.
