The cell and gene therapies transforming rare disease treatment

This Rare Disease Day (28 February 2026), we explore how cell and gene therapies are offering new hope for patients.  Rare diseases, defined as conditions affecting fewer than 1 in 2,000 people, are anything but rare when you consider the numbers. Over 6,000 rare diseases have been identified, collectively impacting around 300 million people worldwide, which is roughly 3.5–5.9% of the global population. These conditions are often genetic, with 72% stemming from inherited mutations, and 70% of cases begin in childhood. For many patients, treatment options have historically been limited, with only around 5% having a licensed treatment, but cell...