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 Venter's privately held company Synthetic Genomics
Inc on Tuesday said it has entered a multiyear deal with United
Therapeutics' Lung Biotechnology Inc to develop the so-called
humanized pig organs.
The venture is intended to advance United Therapeutics' efforts to
develop replacement organs grown in genetically altered pigs.
According to the companies, about 400,000 people in the United
States die each year from various forms of lung disease, and only
2,000 people are saved with a lung transplant.
Prior efforts to use animal organs in people in need of a
transplant, known as xenotransplantation, have failed because of
differences in the genome that caused organ rejection and blood
clots.
“Our new collaboration with Synthetic Genomics is huge for
accelerating our efforts to cure end-stage lung disease," Martine
Rothblatt, chairman and chief executive officer of
Silver Spring, Maryland-based United Therapeutics, said in a
statement.
Humans, pigs and most other mammals share about 90 percent of the
same genes. What Venter's team will do is to determine which aspects
of the pig genome need to be altered to make porcine lungs
compatible with humans, avoiding the rejection response that occurs
even in human-to-human transplants.
"We're going to start with generating a brand new super-accurate
sequence of the pig genome, and then go through in detail and
compare it to the human genome," Venter, the founder and chief
executive of Synthetic Genomics Inc, said in a telephone interview.
"The goal is to go in and edit, and where necessary, rewrite using
our synthetic genomic tools, the pig genes that seem to be
associated with immune responses," said Venter, who is best known
for his role in mapping the human genome over a decade ago and who
created synthetic life in 2010.
"We want to get it so there is no acute or chronic rejection," he
said.
Venter's team is tasked with editing and rewriting the pig genome
and providing the United Therapeutics group with a series of altered
cells. United Therapeutics will take those cells and transplant them
into pig eggs, generating embryos that develop and are born with
humanized lungs.
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If all goes well, Venter thinks his team will be able to deliver
the cells in a few years. Testing the humanized organs in clinical
trials to ensure they are safe in people will take many more years.
Lungs are the hardest organ to transplant because they are so
delicate in structure, Venter says. So, if the team succeeds in
developing humanized pig lungs, hearts and kidneys from these
animals may also prove to be suitable for human transplantation.
As part of the agreement, Lung Biotechnology will take a $50 million
stake in La Jolla, California-based Synthetic Genomics, which also
will receive royalties and milestone incentives from the development
and commercialization of the organs.
Venter admits that just five years ago, the venture would have
sounded like science fiction. But several research teams are working
on the use of genetically altered pig body parts to help improve the
supply of transplant organs.
Last week, researchers at the National Heart, Lung, and Blood
Institute reported at the American Association for Thoracic Surgery
meeting in Toronto that they grafted a genetically altered pig heart
into the abdomen of a baboon and kept it functioning, aided by the
baboon's natural heart, for more than a year.
(Editing by Mohammad Zargham)
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