'This is the time to double down': Top Vertex executives defend research plan as pressure builds

Three months ago, Vertex Pharmaceuticals shared news that investors were eagerly expecting.

A drug the company developed for a rare genetic disease appeared to work as intended, executives said, boosting levels of a key protein without harming patients in a small trial. The finding meant Vertex might eventually be able to pioneer a new type of medicine for a condition with no effective treatments, much as it did for cystic fibrosis.