The Kendall Square biotech boom started nearly a half-century ago, with a small number of scientists who had big ideas. In the decades since, the sector has grown dramatically, now employing more than 106,000 people across the state. These range from startups—think a couple of postdocs at an incubator such as Cambridge’s LabCentral—to multibillion-dollar giants such as Moderna and Vertex Pharmaceuticals. Meanwhile, the vibrant sector here—perhaps rivaled globally only by San Francisco’s—has attracted branches of giants from overseas, such as Takeda Pharmaceutical and Eisai of Japan, and Novartis of Switzerland.
Whatever the company, whatever the approach, the goals are largely the same: to make life better and healthier through science. Here, we examine 12 diseases and conditions—ranging from cancer to chronic pain, Alzheimer’s to postpartum depression—and the innovative companies working on solutions. Some of those treatments may be years away or never end up making it over the hurdles of testing and approval at all. Some could be available in a matter of months.
Vertex Pharmaceuticals is Massachusetts’ most valuable biotech company, with a market capitalization of $75 billion. It achieved that success largely by selling four lifesaving (and expensive) drugs for cystic fibrosis, a rare genetic disease that causes problems with breathing and digestion.
Now Vertex is running multiple late-stage trials on a drug to tame an all-too-common malady: pain. There’s an enormous demand for new pain relievers. Over-the-counter drugs such as aspirin, acetaminophen, ibuprofen, and naproxen were invented generations ago. They are only moderately effective at treating severe pain and can cause stomach or liver problems. Opioids, meanwhile, date back thousands of years. While often highly effective, they have fueled an epidemic of addiction.
Vertex is testing a potent experimental painkiller that doesn’t rely on opioids and is nonaddictive. In a mid-stage study, the pill, dubbed VX-548, significantly reduced acute pain in people recovering from recent surgeries (such as bunion removals and tummy tucks). It’s now being tested in three late-stage trials on acute pain. Opioids operate in the brain and spinal cord to block pain but pose a high risk for drug abuse, a phenomenon of the central nervous system. In contrast, Vertex’s drug inhibits pain signals in peripheral sensory nerves outside the brain and spinal cord, without the potential for abuse, according to the company.
