Rare disease drug discovery: the technologies driving ‘orphan’ therapeutics forward

Rare diseases have long posed a challenge in the field of medicine. Limited treatment options and diagnostic hurdles mean patient prognosis is often not positive; however, some groundbreaking technologies are offering hope of treatment to the small number of people affected by these so-called ‘orphan’ diseases. This Rare Disease Day, we explore how innovations like AI and gene editing are changing the game in rare disease research by advancing drug discovery and development. Rare diseases, as you might expect, are incredibly uncommon, affecting fewer than one in 2000 people. They are often associated with impaired quality of life and reduced...