At Erasmus MC, researchers and clinicians are working on new treatment options for rare diseases, including drug repurposing and cell and gene therapy. This meeting focuses on connection and exchange. Using several practical examples, we will explore opportunities and challenges in the different stages of this process. We will also discuss how academic innovations can reach patients in an affordable and sustainable way, and what types of support are available for this — including from RARE-NL. RARE-NL is a national hub within the FAST platform (Centre for Future Affordable & Sustainable Therapy Development), which will also encompass the emerging ATMP-NL hub.