Advancing Rare Disease & Orphan Therapy Innovation in the Middle East

The Middle East is rapidly becoming a hub for rare disease and orphan therapy innovation, driven by high prevalence of inherited disorders, ambitious national genomics programs, and a growing life sciences ecosystem. This session explores how researchers, hospitals, and biotech companies are developing novel diagnostics, gene and cell therapies, and precision medicine solutions tailored to the region's unique genetic landscape. Participants will hear how local innovation is being accelerated through initiatives such as the Saudi Human Genome Program, expanded newborn screening, and public'private collaborations that bring together academic labs, biotech start-ups, and multinational pharma. From molecular diagnostics platforms to early-stage orphan therapy pipelines, this session highlights the breakthroughs that are positioning the Middle East as a regional leader in rare-disease research and therapy development. Key takeaways: Insights into cutting-edge rare disease diagnostics and precision medicine programs in the region How gene and cell therapy innovation is being adapted to local genetic profiles Examples of public'private partnerships driving orphan therapy research and development Understanding how Middle Eastern initiatives are creating scalable models for rare-disease innovation