Biotechnology companies eGenesis and United Therapeutics Corporation have reported that the FDA greenlit their clinical trials of genetically modified pig kidney transplants last month—the first two ever approved.
It’s an effort to address a sad reality: About 90,000 people are waiting for kidneys in the US, and 11 on the list die every day, the United Network for Organ Sharing estimates.
There are a lot of potential solutions being thrown around, but Robert Montgomery, who directs the NYU Langone Transplant Institute and had his own life saved by a heart transplant, argued that the current organ donation system is a “broken paradigm.” If these trials lead to widely available pig organs, that will be the answer, he said at the Lake Nona Impact Forum in Orlando, Florida, on Feb. 27.
“I really devoted my career to trying to make incremental improvements in the [organ transplant] system, but the system will never be able to deliver enough organs. And this is the future. This is the hope,” Montgomery said.
The science
EGenesis has engineered its donor pigs with 69 genetic edits, including 59 tweaks to remove viruses, the New York Times reported on Feb. 3.
According to a Feb. 7 press release, the company is currently conducting a three-participant trial done under the FDA’s expanded access pathway, aka the compassionate use pathway, a way to test out a Hail Mary for people who are severely ill.
EGenesis also announced in the release a successful kidney transplant for the first trial patient. This is the sixth patient to get a genetically modified pig organ transplant.
“This procedure is more than a scientific milestone—it represents a new frontier in medicine,” Michael Curtis, CEO of eGenesis, said in the release. “We stand at the beginning of a future where organ shortages may no longer dictate patient outcomes.”
There’s a lot of excitement about eGenesis’s gutsy gambit: The company raised $191 million Series D financing last September for this trial, led by Lux Capital.
If the biotech company’s name sounds familiar, it could be because eGenesis also did the first-ever successful kidney xenotransplantation—a transplant from a genetically modified animal to a human—in 2024. Though that patient died 51 days later, experts involved in the transplant previously told Healthcare Brew that there was “no indication” the pig kidney contributed to his death.
United Therapeutics is taking a slightly different approach with a kidney from a 10 gene-edited source pig. That trial, set to kick off mid-2025, will start with six people and expand up to 50 if things go well, according to a Feb. 3 press release.
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The trial is open to people ages 55–70 who have been diagnosed with kidney failure, have been on dialysis for at least six months, and are either ineligible for a conventional transplant for medical reasons or are on the waitlist but aren’t expected to receive a transplant within five years, per the release.
“Clearance of our [Investigational New Drug application] for this first-ever clinical trial of a xenokidney represents a significant step forward in our relentless mission to expand the availability of transplantable organs,” Leigh Peterson, EVP of product development and xenotransplantation at United Therapeutics, said in the release.
Skeptics squeal
The procedure still has its critics, however.
According to the New York Times, there are some concerns about the low risk that the procedure could allow pig pathogens to “spill over” into the human population. And medical ethicists have said patients may be unable to fully consent because there are so many unknown risks.
Andy Howard, co-chair of nonprofit advocacy group the Kidney Transplant Collaborative, told Healthcare Brew that the trials are an “exciting step forward,” but other pathways—like living kidney donation—are a more realistic way to solve this problem right now.
“While this innovation holds promise, widespread clinical use is likely at least a decade away, and patients on the waitlist don’t have that kind of time,” he said.
Towana Looney, the longest-living recipient of a genetically engineered pig kidney, is proud of her decision, she said at the Lake Nona Impact Forum.
The 53-year-old grandmother became the fifth person to get a pig heart or kidney transplant on Nov. 25. The first four patients had died within months of transplantation, and her family was afraid she could meet the same fate.
But she didn’t have many other options. She had been on dialysis for nearly nine years and was unlikely to become eligible for a conventional kidney transplant because she had sky-high levels of antibodies that could reject most donor kidneys, Montgomery, who led Looney’s transplant at NYU Langone Health, said at the Lake Nona Impact Forum.
“If you have a family member that’s on dialysis or has a kidney disease, tell them to step out on faith…Don’t be afraid,” Looney said.