13 min read
PCSK9: The Gene That Proved a Drug Would Work Before It Existed
How a genetic discovery in 2003 launched a $5 billion market.
In 2006, researchers found people born with broken copies of PCSK9 had very low cholesterol and almost no heart attacks. They were healthy. That was the signal. Four drugs across three different modalities now target the same gene - a combined market exceeding $5 billion in 2025.