Louis and the founder of Rare Genomics, a nonprofit organization that designs treatments for rare childhood diseases based on individual genetic analysis, “we have drugs that target specific cancer mutations. Examples include
Gleevec, Zelboraf, and Xalkori. Vertex,” a pharmaceutical company based in Massachusetts, “has famously made a drug for cystic-fibrosis patients with a particular mutation. The genetic targeting of individuals is a little farther out.