Gene therapy for deafness given away free in statement on industry purpose
Regeneron Pharmaceuticals co-founder and chief scientific officer George Yancopoulos is more comfortable in a leather jacket than a suit, according to the Journal profile. His own team talked him into wearing one to an April meeting with President Trump, though he paired it with a colorful tie hand-painted by Nobel laureate Wally Gilbert.
The meeting was organized in part to tout Regeneron’s latest invention: a gene therapy that restores hearing to children born with a rare genetic deafness. The condition affects fewer than 50 newborns a year in the U.S. At Yancopoulos’s urging, Regeneron decided to give the therapy away free.
“We did it to make a statement,” Yancopoulos, 66, told the Journal. “We want to remind people that when people are sick, you pray for a miracle. And where do they come from? They come from our industry.”
Nearly four decades since he co-founded Regeneron, Yancopoulos has turned the company into one of the world’s most valuable drug companies, with a market capitalization of roughly $70 billion. He has done so by repeatedly pioneering technologies that opened up entirely new classes of blockbuster drugs, the Journal reported.
Regeneron’s flagship eye drug, Eylea, has preserved the sight of millions of people with a common cause of blindness and generated roughly 80% of the company’s revenue at its peak. It is now facing increasing competition, the Journal reported. Another star drug, Dupixent, will begin to lose patent protection in the early 2030s. Since the start of last year, two of Regeneron’s late-stage drugs have failed in clinical trials, sending the stock down more than 15% year-to-date.
Many drug companies in Regeneron’s position would go shopping for a smaller biotech’s late-stage drug. Regeneron is in a stronger position than many other companies to do deals, with more than $18 billion of cash on its balance sheet. Yancopoulos is betting on Regeneron’s science instead. The company has the broadest pipeline in its history, with about 60 drugs under development. The pipeline spans oncology, neurodegenerative diseases such as Alzheimer’s and Parkinson’s, and obesity.
“It may be hard to get another $10 billion asset, but if we get three or four smaller assets, it actually may be as good,” Yancopoulos said. “It’s even healthier for a company in the long term to have multiple programs.”
Yancopoulos’s scientific career traces back to age 13, when his grandmother developed dementia. The family was living in the Woodside neighborhood in Queens, N.Y., where his parents had immigrated from Greece. The teenager wanted to cure his grandmother. An advanced science student at Bronx High School of Science, he tried to regenerate neurons destroyed by disease as a school science project. When that proved too difficult, he switched to experimenting with single-celled organisms. His work came in ninth in the 1976 Westinghouse Science Talent Search, the prestigious competition that Regeneron has sponsored since 2016.
His idea also sparked the name of the company he would go on to co-found, the Journal reported.
When Yancopoulos told his father — a Greek military veteran who later became a salesman in the U.S. — that he wanted to become a scientist, his father steered him toward the private sector. He handed him a newspaper clipping about Roy Vagelos, the Greek American scientist leading research at Merck. Years later, after earning his bachelor’s degree, MD and Ph.D. at Columbia University and staying on as a junior faculty member, Yancopoulos won a $2.5 million faculty grant. Curing diseases, his father told him, would take far more money than government grants could provide.
By the late 1980s, Yancopoulos was an up-and-coming molecular biologist at Columbia’s campus in northern Manhattan. A neurologist named Leonard Schleifer courted him for more than a year to team up on a biotech venture that Schleifer was starting in a one-bedroom apartment at Cornell University’s medical resident housing. Yancopoulos and Schleifer toured real estate across New Jersey, Long Island and Manhattan before they finally settled on an industrial park in Tarrytown, N.Y., for Regeneron.
Today, Schleifer still runs Regeneron’s business side, and Yancopoulos runs the science — a partnership that has outlasted the industry’s average executive tenure several times over.
“We fight like cats and dogs,” Schleifer told the Journal, “until he points out that he’s right and I’m wrong.”
Yancopoulos’s biggest wins have come from being years ahead of everyone else. Eylea received regulatory approval in 2011; the next biggest competitor was approved in 2022. Dupixent, first approved in 2017, was nearly five years ahead of the next drug and is now the most widely prescribed antibody on the planet, treating nine different allergic diseases, according to the Journal.
It is getting harder to stay ahead of rivals. Chinese biotechs are moving quickly to replicate successful science — a threat to the entire industry but one that cuts to the core of what Regeneron has been able to capitalize on. Yancopoulos knows it is unlikely he’ll be able to fend off competition for very long, even with unique new discoveries, the Journal reported.
Instead of betting on one more mega-blockbuster, Regeneron’s research spans multiple therapeutic areas. The company’s newly completed preclinical manufacturing laboratory complex in Tarrytown reveals the scale of that effort. The nearly autonomous facility allows scientists to grow mammalian cells from genetically humanized mice remotely and monitor stainless-steel bioreactors in real time. It is a long way from where the company started, with Yancopoulos growing his first cells in one-liter bottles checked and fed by hand every 12 hours on homemade wooden roller machines.
Nowhere is Yancopoulos’s strategy clearer than in obesity, where dozens of companies are racing to launch their own versions of GLP-1 drugs. Yancopoulos, the Journal reported, hates the copycat model.
“Do we need 168 GLPs? Do we need somebody to make five new versions of Dupixent so they can get a little bit of our business? It just doesn’t seem right,” Yancopoulos said.
Yancopoulos is chasing a different angle: Patients on GLP-1s can lose significant amounts of muscle along with fat, a particular risk for older adults who need the strength to stay independent. Regeneron has been developing a pipeline of muscle-preservation drugs that block the body’s natural atrophy systems. By pairing these therapies with weight-loss medications, Yancopoulos hopes to focus on the quality of weight reduction so patients get stronger rather than just thinner.
Yancopoulos is also using Regeneron’s in-house genetic discoveries to tackle obesity. By mining their trove of human DNA, Regeneron scientists discovered what he calls the “laziness gene,” which dictates whether a person is inherently sedentary. The company is developing a drug it hopes will turn off that genetic switch, allowing patients to naturally burn more calories.
“The hardest thing that science and arguably humanity tries to do is to come up with breakthroughs and important new medicines,” Yancopoulos said. “Regeneron has managed to deliver time and time again.”