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Biotech and Pharma

CEO's message to investors: My biotech is like Google before it was big

Medical 'moonshot': Manufactured organs

The founder of United Therapeutics told CNBC on Tuesday that investors should get into her biotech firm now, before it grows to Google-like proportions.

"You got to look around the corner," said Martine Rothblatt, who earlier in her career started Sirius Radio, which later merged with XM to form Sirius XM. "Today our business is all about pharmaceuticals. But in the 2020s, I think you're going to see a transition ... to regenerative medicine — tissues, beyond cell therapies, and then whole organs."

In collaboration with genome sequencing pioneer J. Craig Venter and his firm Synthetic Genomics, United Therapeutics is working on lab-grown organs.

"Our company is well positioned. We [also] own the U.S. arm of the Dolly the Sheep company, The Roslin Institute," Rothblatt said on "Squawk Box." Dolly was the world's first mammal to be cloned from an adult cell.

"All [organs] are readily doable," she said. "In fact, they're already being tested in baboons, which is a model for the human. We have hearts at [National Institutes of Health] NIH now. Our hearts last over two years in the baboons. "

Rothblatt started United Therapeutics in 1996 to help find a cure for her daughter's pulmonary hypertension. The firm currently has four drugs on the market to treat the disease.

Along with other biotech stocks, United Therapeutics has had a tough year, down nearly 30 percent in 2016. But that's nearly double the decline of the iShares Nasdaq Biotechnology ETF. United Therapeutics has market value of nearly $5 billion.

The stock price notwithstanding, Rothblatt see big things ahead. "There are some far-sighted investors that say, 'Hey, I have a chance to get in on ... [an] Amgen before it was Amgen; or ... Google before it was big.' "

Admitting it's early days for lab-grown organs, Rothblatt maintained a commercial product might not be that far off.

"We'll have the first transplantable organs in patients before the end of this decade, before the end of 2019," she predicted. "[Then] I would say FDA approval around 2023."

Rothblatt was thrust into the spotlight in 2014, after it was revealed she was the highest paid female executive in America, with a 2013 pay package of $38 million.

At the time, Rothblatt told Fortune she felt awkward from the attention because she's only been a women for half of her life. In 1994, she underwent sex-reassignment surgery.

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