- United Therapeutics CEO Martine Rothblatt is a trailblazer in drug development for rare diseases.
- She is now developing technology to tackle the organ shortage in America.
- Her goal: to use genetic engineering to make pig organs viable transplant in humans.
- Through its subsidiary Lung Biotechnology, it has forged an agreement to operate a lung restoration facility at the Mayo Clinic in Jacksonville, Florida by year-end.
Sometimes a Eureka moment sparks a breakthrough in innovation; other times it's a rush to solve a crisis plaguing society — and the ones you love. That was the case for Martine Rothblatt, CEO of United Therapeutics, a Maryland-based company she founded in 1996, and former CEO of GeoStar and creator of SiriusXM Radio.
Over the years, Rothblatt has become something of a Renaissance figure in the world of drug development after she invented a drug to cure a rare and fatal lung disease that struck her youngest daughter. Now she has a keen interest in growing transplantable pig organs that can be used in humans — especially those who need lung transplants. The world she envisions: an organ manufacturing industry where organs can be grabbed like spare parts to save lives.
United Therapeutics created a subsidiary for its organ effort called Lung Biotechnology Public Benefit Corp. It is involved in organ delivery, lung regeneration to boost the supply of lung organs for transplants, and xenotransplantation (cross-species transplantation).
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The company's efforts may sound futuristic, but it comes at a pivotal time when demand for organs is growing and advancements in gene-editing techniques could make it possible for cross-species transplantation. There are 114,000 sick patients on the organ transplant list in the U.S., but about 8,000 people die every year waiting for the organs they need, according to data from the United Network for Organ Sharing.
Through Lung Biotechnology, it has forged an agreement to operate a lung restoration facility at the Mayo Clinic in Jacksonville, Florida, by year-end. The facility will use ex vivo lung perfusion machines to assess and treat donor lungs prior to transplant. It will be a game-changer for people with lung disease.
But the organ shortage is just the latest medical enigma Rothblatt is trying to solve. She is known for cracking the code on a rare lung disease called pulmonary arterial hypertension (PAH) by finding the molecule used to develop the drug Remodulin, which was approved by the FDA in 2002. Remodulin slows the worsening of symptoms and is safe and effective in children. Today that medicine is used by thousands of patients.
That effort took years, but it was a quest Rothblatt championed in an effort to save her daughter's life. PAH is a form of high blood pressure in the lungs that over time obstructs the arteries that go from your lungs to your heart.
It started when Rothblatt contacted drug companies after her daughter's diagnosis and found that drug development for rare diseases was paltry at best. "Big Pharma was not willing to spend $1 billion on R&D for a drug that could help a small patient population of about 2,000 in the U.S," she recalls.
"Once I understood what the game was, I knew I had to find a solution myself," she recalls, noting her passion and interests were never in biology and bioscience but in physics and space technology.
"To succeed with such an effort, you have to feel that the success of your project or your invention is even more important than your own life," Rothblatt says.
That passion for disruption in medicine, as well as other fields, defines the transgender CEO and entrepreneur. And she is no stranger to adversity or naysayers who think her pursuits are unattainable. "I think my critics first looked at me in a Darwinian selection sense that though of my technology like mutant technology."
Today her company's milestones speak for themselves. As she told Jim Cramer, the host of CNBC's "Mad Money": "[United Therapeutics] prides itself in being different, and the corporate mantra is to identify the corridors of indifference where the pharmaceutical companies don't go, and run like hell down those corridors."
While doing so, Rothblatt's daughter has grown into a healthy adult.
But United Therapeutics has not been immune to the problems facing many biotech and drug companies, including the threat of generic competition and trouble related to high brand prices. The company has been the target of a lawsuit by Sandoz, a division of Novartis, alleging that it unfairly thwarted generic competition. Currently, Remodulin is one of the most expensive drugs covered by Medicare, costing patients an average of $144,000 annually.