Pay attention.  I am going to tell you a story about a most incredible woman.

While in college, this person made a trip to the Seychelles.  And, was amazed by the NASA satellite tracking system there.  So much so that when this person went back to UCLA, the thesis topic chosen covered international direct broadcast satellites.  And, then, this person went on to earn a JD (Juris Doctor) and an MBA.

This regulatory attorney became an entrepreneur in the field of broadband/satellite technology. A decade or so later, this consultant helped folks commercialize satellite technology. Then, the founding of  GeoStar (car navigation) and Sirius (satellite radio),  a healthy marriage with Bina Aspen, and four great kids.

Well, one of those kids (Jenesis) had an affliction- pulmonary arterial hypertension.  Which meant full respiratory functionality wasn’t in the cards.   So, this person read and studied everything and anything that could be found about this condition.

Using some of the money garnered from Sirius’ public offering, this person wanted to fund a grant program, with Jenesis’ pediatric cardiologist serving on the grant review committee.

That’s how  where this person became aware of a drug owned by Burroughs Wellcome (which had just been subsumed by Glaxo).   It was capable to relaxing the arterial smooth muscle tissues- which means it could be possible to restore the blood flow for those with pulmonary arterial hypertension.

Well, Glaxo had a rule.  It would not continue research into any drug that would not be serving a $ 1 billion annual market.  So, it killed the project- and retired the scientific staff involved with its development.

Glaxo told this person that their decision was final- and it had zero inclination to license the product to a non-profit that lacked clinical expertise- and also had no employees.

You know what this person did?  Started a drug company and hired the recently retired scientists to populate the firm. (The chances of success, by the way, were considered slim-to-none.)   But a year later, this person had added a team of chemists that allowed this molecule to become a mass-produced drug.  No simple task- the production scheme involved 23 separate steps.

Glaxo now gets 10% of the profits from Remodulin, the drug in question.  Oh, and that drug generates billions in sales.

(The company’s new drug, Tyvaso DPI [treprostinil], an inhaled drug that treats PAH is awaiting FDA approval.  The FDA is concerned by an inspection issue at the [third party] firm that performs analytic service for the drug.)

About the time this person found about Jenesis’ condition, this person had begun a most unusual transition.  Sexual conversion to become a woman. Her name is Martine Rothblatt.  (That’s why I kept using “this person” until now.)

You might think that’s the end of the story.  Boy, are you wrong!

To ensure Martine would understand clinical trial systems (critical for any successful biotech), she understood that matriculation at Barts and the London School of Medicine and Dentistry was required.  Her Ph.D. thesis? Animal to human transplantation  (Xenotransplantion).  Which became the book published in 2003, Your Life or Mine: How Geoethics Can Resolve the Conflict Between Public and Private Interests in Xenotransplantation.

Now, a little aside.  You have been reading my blogs right?  If so, you should recall a very recent piece about xenotransplantation.   Yup, that was about a division of Martine’s company, United Therapeutics.

This is not a small firm- it’s worth somewhere in the neighborhood of $ 8 billion.  Supplying about 100,000 folks with that drug, Remodulin.  And, Dr. Rothblatt is the highest paid female executive in the US.  Oh- and Jenesis (now in her 30s) is an employee of United Therapeutics, too.  Not to mention the United Therapeutics is the only publicly traded company to be a public benefit corporation (a “B” corporation).

By now, you probably won’t be surprised that to populate Revivicor, the United Therapeutics division that produces the pig supplied organs, Rothblatt hired David Ayres and his team.  Whose Ayres?  What team?  The team that developed Dolly, that lamb clone that made all the headlines back in the 1990s.

For the pigs, the team has removed four genes and supplemented the DNA makeup with six human genes.  That makes the pig ‘human-compatible’.  And, the team plans to produce kidneys (already reported here), hearts, and lungs for human transplantation.

The driving force behind Dr. Rothblatt’s quest for xenotransplantation?  She knows that it’s just a matter of time before Remodulin stops keeping her daughter, Jenesis, alive.  And, if you think kidney transplants are tough to come by, you haven’t checked on lung transplantation.

The quest to have these GalSafe pigs yield not just kidney and heart organs- but lungs.  To ensure Jenesis has a chance for her own genesis.