Rare disease research is entering a transformative era. Advances in precision medicine, in vivo gene editing and novel therapeutic modalities are opening unprecedented opportunities to address conditions that have long lacked effective treatments. However, scientific innovation alone is not sufficient. Translating breakthrough discoveries into accessible therapies requires overcoming significant challenges in clinical development, manufacturing, regulation and healthcare implementation.
This round table will explore the opportunities and barriers shaping the next generation of rare disease therapies, with particular attention to ophthalmology and central nervous system (CNS) disorders. Experts from biotechnology, pharmaceutical industry and translational research will discuss how emerging technologies—including in vivo gene editing and other precision therapeutic approaches—are changing treatment paradigms, while examining practical issues such as innovative clinical trial designs for ultra-rare populations, scalability of development programmes, biomarker-driven patient selection, and the path towards sustainable access.
The discussion will highlight how collaboration between academia, biotech, industry and healthcare systems can accelerate the delivery of innovative therapies to patients, positioning Spain and Europe as key contributors to the future of rare disease innovation.
Potential speakers
