AI promises a new future of drug discovery for rare diseases

Despite affecting over 400 million people around the globe, rare diseases have largely been overlooked by traditional drug discovery methods. This is because traditional model often prioritise disease areas where there is a greater understanding of the basic underlying biology and which, in turn, generally come from the larger patient populations associated with more common diseases. This is part of the reason why 95% of rare conditions lack an approved treatment today. Promisingly though, rare disease companies are working hard to not only pioneer individual therapies for conditions, but actually pioneer entire new approaches to the discovery of therapies for rare diseases. This work has been made easier thanks to landmark legislation like the Orphan Drug Act of 1983, but it’s also been drastically accelerated thanks to the evolution of artificial intelligence (AI). Indeed, new technologies like AI are allowing novel treatments to be designed, developed and delivered more quickly and on a scale never before seen in the industry, thereby offering hope to millions of patients in need and ushering in a new era of drug discovery.