Key to compatibility | Experts look at altering pig genome to prevent organ rejection and blood clots
CHICAGO (Reuters) — Genome pioneer J. Craig Venter is teaming up with a unit of United Therapeutics Corp. to develop pig lungs that have been genetically modified to be compatible with humans.
The feat, if successful, could address the urgent need for transplant organs for people with end-stage lung disease.
Venter’s privately held company, Synthetic Genomics Inc., said it has entered a multiyear deal with United Therapeutics’ Lung Biotechnology Inc. to develop the humanized pig organs.
The venture is intended to advance United Therapeutics’ efforts to develop replacement organs grown in GM pigs. According to the companies, 400,000 people in the United States die each year from lung disease, and only 2,000 people are saved with a lung transplant.
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Previous 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,” said Martine Rothblatt, chief executive officer of United Therapeutics.
Humans, pigs and most other mammals share 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. This will avoid the rejection response that occurs even in human-to-human transplants.
“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 more than a decade ago and who created synthetic life in 2010.
“We want to get it so there is no acute or chronic rejection.”
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 transplant those cells into pig eggs, generating embryos that develop and are born with humanized lungs.
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 said. Hearts and kidneys from these animals may also prove to be suitable for human transplantation If the team succeeds in developing humanized pig lungs.
As part of the agreement, Lung Biotechnology will take a $50 million stake in Synthetic Genomics, which also will receive royalties and milestone incentives from the development and commercialization of the organs.
Venter said the venture would have sounded like science fiction just five years ago. However, several research teams are working on the use of GM pig body parts to help improve the supply of transplant organs.
Researchers at the National Heart, Lung, and Blood Institute recently reported at the American Association for Thoracic Surgery meeting in Toronto that they grafted a GM pig heart into the abdomen of a baboon and kept it functioning, aided by the baboon’s natural heart, for more than a year.
