Vertex Pharmaceuticals

A new era of personalized medicine began last fall when the U.S. and other countries gave the first-ever approvals to a treatment based on CRISPR gene editing: Casgevy, developed by Vertex and CRISPR Therapeutics, treats sickle cell, a genetic disorder that affects some 20 million globally. The pioneering treatment is expensive (the list price is $2.2 million), and may not be initially feasible in sub-Saharan Africa, where about 75% of people with sickle cell live. But by April, the first patients had started the treatment process. Vertex expects revenues to top $10 billion in 2024, largely powered by another remarkable drug, cystic fibrosis treatment Trikafta, which got FDA approval in 2019 and has transformed patients’ lives. CEO Reshma Kewalramani calls the historic sickle-cell breakthrough a beginning. “We are on the cusp of moving from…taking pills by mouth every day,” she says, “to potentially one-and-done therapies, where you have a procedure once and hopefully benefit for life.”