Last year, the European Commission approved a record 55 therapies containing a new active substance, including 21 medicines for rare diseases. Among them were a number of gene therapies for indications such as cancer and hemophilia.

If a clinical trial is positive, with safety and efficacy that trounces the competition, why be coy about the details?

AnGes has terminated R&D work on a DNA vaccine for COVID-19 and will now look at intranasal candidates with a US partner. Although the original project received funding from the Japanese government, a spike in related costs caused the Japanese firm's net loss to triple last year, and the decision represents the latest in a string of pipeline setbacks.

Despite broad interest, real-world evidence of efficacy in FDA decisions remains largely limited to orphan diseases and natural history, a Pink Sheet analysis shows.