A human heart created from a genetically modified pig.
Diseased lungs cleaned up and restored for human transplants.
Custom-made, 3-D printed organs.
This may sound like the stuff of Stephen King novels. But these products are part of a budding organ manufacturing industry, where an organ transplant will be more like grabbing a spare car part than waiting for a life-saving lottery. It’s a future that Martine Rothblatt, one of Maryland's most recognizable biotech CEOs, is working to make a reality at her company, Silver Spring’s United Therapeutics Corp. — and she predicts this science fiction could start to become scientific fact in the next five years.
Transforming organ transplantation is just the latest moonshot for a serial entrepreneur and futurist who pioneered satellite communications by founding Sirius Radio, built a $1.5 billion biotech to invent the pulmonary hypertension drug that would save her daughter’s life and, in a side project, has been working to achieve immortality through digital doppelgangers. Her newest incarnation of United Therapeutics could boost a company facing crushing drug patent losses. In doing so, she’s hoping to make Maryland a leader in the emerging organ development industry and, ultimately, save millions of lives.
It comes down to a question she asked in 2015 at a TED media organization discussion: “Just like we keep cars and planes and buildings going forever with an unlimited supply of building and machine parts, why can’t we create an unlimited supply of transplantable organs to keep people living indefinitely?”
She’s determined to wrestle that “engineering problem,” as she calls it, to the ground. Even as Wall Street closely watches whether United Therapeutics can defend against impending generic competition following patent expirations on its flagship drugs, the company has been unveiling bit by bit — through Rothblatt’s public remarks, new partnership announcements and federal regulatory filings — the breadth of its more ambitious, long-term play to conquer the global organ shortage. It’s already restoring diseased donor lungs that would traditionally have gone to waste, while racing to produce animal organs to transplant into humans.
United Therapeutics, its leaders and its subsidiaries declined to comment for this story, opting to hold off until clinical testing of organ manufacturing is further along. Even Wall Street is reticent, with many analysts saying they are not yet comfortable addressing the transplant side of the company’s business.
But it’s a topic Rothblatt has increasingly discussed since mid-2015 in the company’s quarterly conference calls and financial reports. Indeed, in its most recent annual report, filed with the Securities and Exchange Commission, the biotech divulged that it expects federal approval to use one of its manufactured organs sometime between 2022 and 2025.
“In the next 10 to 20 years, Maryland will be known as the state that pioneered the concept of organ manufacturing,” Rothblatt told a ballroom of biotech insiders while accepting a lifetime achievement award from the Tech Council of Maryland in 2016. “These things people thought were not manufacturable are manufacturable.”
The business of replacement parts
As Rothblatt described her organ manufacturing plan to business leaders at a Leadership Greater Washington event in December, stunned gasps and murmurs reverberated through the audience.
“We are going to be able to say to a patient suffering from end-stage heart disease or liver disease or lung disease,” Rothblatt said, that we’ll “provide you a replacement organ that matches your DNA so you won’t have to take any immunosuppressants.”
In essence, she said, the company is working to offer “custom-designed” organ transplants.
That future is still a way off, but it is one of several approaches where United Therapeutics has thrown its weight — and funding. Of the company’s $99 million increase in research and development spending last year, $36.3 million flowed to increased preclinical work on organ manufacturing projects.
Its subsidiary, Lung Biotechnology Public Benefit Corp. in Silver Spring, is heading up efforts to address current shortages of lung and other organs by delaying the need for them or expanding the supply. It specializes in what’s called ex vivo lung perfusion, a process that preserves and restores human donor lungs to make more of them viable for transplantation. The subsidiary has said its technology can keep lungs outside of the body for up to 22 hours, considerably longer than the six hours most of its competitors can manage. “We’re taking lungs no one else wants and making them transplantable 65 percent of the time,” Rich Pietroski, president of Lung Biotechnology, told the Washington Business Journal in 2016.