Serial entrepreneur Martine Rothblatt is making a bold bet on genetic modification.
Martine Rothblatt has a knack for inventing or backing technologies that initially seem far-out but ultimately end up embedded in people’s lives. In 1990 she founded a company that was the first to seek to use frequencies assigned to television broadcasters for a satellite-based radio service — a move that was pooh-poohed by the radio industry (“You’ll have to buy a whole new radio!”). Rothblatt’s company eventually evolved into Sirius XM Radio SIRI , now valued at about $19 billion.
A subsequent venture, United Therapeutics UTHR , aimed to develop a drug therapy for pulmonary arterial hypertension as an alternative to heart and lung transplants. The company’s drugs are leaders in the treatment of the disease. Last year it posted revenue of $1.1 billion.
Not surprisingly, Rothblatt’s latest project is avant-garde and hugely ambitious: United Therapeutics has teamed up with Craig Venter’s Synthetic Genomics, a biotech company based in La Jolla, Calif., to develop pigs with genetically modified organs — for transplantation into humans. The goal? To create an unlimited supply of organs “so nobody ever again has to face the triage of dying on the transplant list,” Rothblatt told Fortune in October.
Animal-to-human organ transplants, known as xenotransplantation, might sound like extraordinary stuff, but Rothblatt has the track record for innovation, the intellectual curiosity, and the deep pockets needed to do pioneering research in this area.
After earning a joint law and business degree from UCLA, she did legal and regulatory work for clients such as NASA and the television broadcast industry before becoming an entrepreneur. At various points in her career, she has gone back to school to study astronomy and medical ethics. She’s also very wealthy. Rothblatt, who is chairman and CEO of United Therapeutics, often ranks among the highest-paid women executives in the U.S., and last year her total compensation was $38.2 million, according to pay consultancy Equilar. “Martine is a superhuman being,” says Liana Moussatos, an analyst at WedBush Securities who follows United Therapeutics. “She’s above the majority of us.” (Rothblatt, born Martin Rothblatt 60 years ago, underwent sex-reassignment surgery in 1994 and speaks openly about transgender issues. She and wife Bina have been married for 32 years and have four children.)
To be sure, xenotransplantation is a far more complicated R&D project than niche-drug development. The body sometimes rejects organs transplanted from its own species, let alone those of the porcine kind. The University of Michigan’s Jeffrey Platt, a professor focusing on the immunobiology of transplantation, acknowledges the rejection issues that xenotransplantation presents, but notes that human-to-human transplantation was considered experimental until the 1960s and not widely applied until the 1980s.
“The National Institutes of Health is extremely conservative. It’s so hard to get funding for anything truly novel,” he says. He’s cautiously excited by United Therapeutics’ foray into genetic modification, largely because it will advance the movement: “Whatever they try will fail, if the past is any guidance. The real question is, Will they keep moving forward? Their success will revolutionize medicine.” And with any luck, Rothblatt will again make the far-out seem mainstream.
This story is from the June 16, 2014 issue of Fortune.