United Therapeutics’ Martine Rothblatt Envisions Abundant Supply of Lung Transplants

illustration of lungs

Martine Rothblatt cofounded satellite radio company Sirius XM in 1990 but has since moved into biotech. The shift was related to her daughter’s diagnosis with pulmonary arterial hypertension (PAH). At the time of the diagnosis, the only treatment was an intravenous drug marketed by GlaxoSmithKline. Rothblatt founded United Therapeutics, which now sells five approved drugs, Adcirca, Orenitram, Remodulin and Tyvaso, all for PAH, and Unituxin for pediatric neuroblastoma.

The company’s subsidiary, Lung Biotechnology, as the name suggests, focuses on various lung diseases. But one of the areas the company is working on is genetically modified pigs whose organs could be manufactured for transplants, including lung transplants. It is approaching the area of transplantable lungs from several different directions.

CNBC points out that in the U.S. alone there are 114,000 sick patients on the organ transplant list, but approximately 8,000 people die waiting for organs to become available. Lung Biotechnology inked a deal with Mayo Clinic in Jacksonville, Florida, to run a lung restoration facility. The agreement was originally signed in mid-2015. It has the goal of significantly increasing the number of lungs available for transplantation by preserving and restoring chosen marginal donor lungs, making them usable for transplants. Construction on the facility began in late 2017 and is opening up this year.

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In 2017, Mayo also signed a deal with Baxter International to develop a new center of excellence for chronic kidney disease at its Florida campus. And earlier this year, Mayo signed a strategic pact with Leidos to accelerate research and development tools and to build a product development ecosystem at the Florida campus.

Part of the deal between Mayo and Lung Biotechnology is to us ex vivo lung perfusion machines to evaluate donor lungs and treat them before transplant. But Rothblatt’s approach to pig organ transplants is different, although it’s not unheard of. The idea is to genetically engineer pigs so they or their organs do not have the genetic biomarkers that trigger organ rejection common to transplant patients.

Rothblatt described this when she spoke at a Leadership Greater Washington event in December 2017, “We are going to be able to say to a patient suffering from end-stage heart disease or live disease or lung disease,” that we will “provide you a replacement organ that matches your DNA so you won’t have to take any immunosuppressants.”

The company has also been working growing organs in the laboratory from scratch using tissue regeneration. In this approach, scientists take pig donor lungs, strip them of their own cells and use the remaining collagen structure as an organ scaffold, which is repopulated with billions of human cells, which completely replaces the pig collagen with human DNA-directed collagen. The result, if it comes together, is essentially completely human lung grown in a laboratory.

Another approach is a deal United Therapeutics and Lung Biotechnology made with Israeli 3D bioprinting company CollPlant last year. United paid CollPlant $5 million upfront with up to $15 million in milestones to supply bio-ink to Lung Biotechnology. CollPlant’s recombinant human collagen (rhCollagen) is grown from tobacco plants engineered with five human genes. The purified collagen can be used as a scaffold for 3D bioprinting solid organs.

At the time, Rothblatt stated, “CollPlant’s extraordinary Israeli technology transforms the tobacco plant that is so associated with lung disease into a collagen-expressing plant that will be essential to the production of an unlimited number of transplantable lungs.”

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