Ana Hidalgo-Simon, Leiden University Medical Center, The Novo Nordisk Foundation Center for Stem Cell Medicine (reNEW)

The dynamic field of translational medicine, particularly in the context of cutting-edge cell and gene therapies, is reshaping the role of academic research centers. This session will discuss optimizing the academic research framework for clinical relevance, focusing on regulatory science, accessibility, stakeholder engagement, and organizational optimization.

Academic institutions are increasingly pressurized to drive the progression from pre-clinical stages to clinical trials, with the potential to achieve market authorization. With the rising interest in academic drug development and the backdrop of healthcare sustainability challenges, there's a growing shift of public funding towards research collaborations focused on clinical applications. However, institutions often lack the specific capacity, knowledge and focus, which are essential for successfully navigating the entire development pathway to market authorization.

The session will explore pivotal areas for researchers in translational medicine, stressing the importance of translatable approaches for research impact. Accessibility, including regulatory navigation and Health Technology Assessment, is vital for successful translation and influences funding and EU legislation debates. Engaging stakeholders strategically is crucial for aligning therapies with clinical goals and patient expectations. Finally, the need for organizational innovation is discussed, highlighting strategies for cross-disciplinary collaboration and rethinking structures to support research's transformation into clinically impactful therapies.

16:00 - 16:10 Ana Hidalgo-Simon, Leiden University Medical Center, Netherlands INTRODUCTION AND SETTING THE SCENE

16:10 - 16:20 Stefan Irion, BlueRock Therapeutics, USA ACCESS TO THE CLINIC: WORKING TOWARDS A SUSTAINABLE HEALTH SYSTEM

16:20 - 16:35 PANEL DISCUSSION: ACCESS TO THE CLINIC Panel Moderator: Ana Hidalgo-Simon, Leiden University Medical Center, Netherlands Panelists: Stefan Irion, BlueRock Therapeutics, USA Chantal van Litsenburg, Leiden University Medical Center, Netherlands Melanio Mauricio, Leiden University Medical Center, Netherlands

16:35 - 16:45 Lisa Ott de Bruin, Leiden University Medical Center, NetherlandsSTRATEGIC STAKEHOLDER ENGAGEMENT

16:45 - 17:00 PANEL DISCUSSION: STRATEGIC STAKEHOLDER ENGAGEMENT Panel Moderator: Ana Hidalgo-Simon, Leiden University Medical Center, Netherlands Panelists: Lisa Ott de Bruin, Leiden University Medical Center, NetherlandsLeanne Rees, Murdoch Children’s Research Institute, Australia Stefan Irion, BlueRock Therapeutics, USA

17:00 - 17:10 Chantal van Litsenburg, Leiden University Medical Center, Netherlands OPTIMIZING THE ORGANIZATIONAL FRAME: CHALLENGES IN A CHANGING ENVIRONMENT

17:10 - 17:25 PANEL DISCUSSION: OPTIMIZING THE ORGANIZATIONAL FRAME Panel Moderator: Ana Hidalgo-Simon, Leiden University Medical Center, Netherlands Panelists: Chantal van Litsenburg, Leiden University Medical Center, Netherlands Lisa Ott de Bruin, Leiden University Medical Center, Netherlands Josefine Rågård Christiansen, University of Copenhagen, Denmark

17:25 - 17:30 Ana Hidalgo-Simon, Leiden University Medical Center, Netherlands CLOSING REMARKS