In a landmark move, the US Food and Drug Administration (FDA) has given biotech company eGenesis the go-ahead to conduct a broader human trial in pig-to-human kidney transplants, marking a significant step forward in addressing the critical organ shortage facing thousands of Americans.
The FDA has given eGenesis its Investigational New Drug (IND) clearance for EGEN-2784, paving the way for a Phase 1/2/3 study evaluating its safety and efficacy over 24 weeks in patients aged 50 or older who are on dialysis and awaiting a kidney transplant. The procedure, known as xenotransplantation – transplanting organs, tissues or cells from one species to another – is seen as the most promising way to meet the need of patients waiting desperately for an organ to become available.
The IND clearance comes as the company reveals two landmark milestones from its ongoing Expanded Access study. The first patient, 67-year-old Tim Andrews, has now been off dialysis for more than seven months, after his EGEN-2784 transplant, making him the longest-living recipient of a genetically engineered pig-derived kidney to date. A second patient, Bill Stewart, 54, received an EGEN-2784 transplant in June and was discharged just a week later. Both patients received transplants at Massachusetts General Hospital (MGH) under highly controlled conditions.
In 2023, Richard Slayman became the first patient to receive a genetically modified pig kidney – and while he died a year later due to heart failure, it was not related to the transplanted organ.
“This is a historic moment for xenotransplantation," said Leonardo V. Riella, M.D., medical director for kidney transplantation at MGH ,who led the clinical study. "To see a patient thriving off dialysis for over seven months with a genetically engineered pig kidney is nothing short of groundbreaking.
“This achievement was made possible by extraordinary collaboration across academia, industry, and government. I want to recognize eGenesis for its cutting-edge gene-editing technology and essential partnership; our MGH transplant team for their clinical excellence; and the FDA for their rigorous and supportive guidance. Additional critical industry partners include Eledon, Apellis, and Karius. Together, we are not only making history – we are reshaping the future of transplantation.”
The new ruling opens up the landmark procedure to more patients experiencing end-stage kidney disease (ESKD).
EGEN-2784 carries three classes of genetic edits: Removal of porcine genes linked to hyper-acute rejection, insertion of seven human transgenes to regulate immune response, inflammation, blood clotting, and complement pathways, and the inactivation of porcine endogenous retroviruses. Basically, these genetic modifications make pig kidneys more compatible with human recipients.
“Clearance of the EGEN-2784 IND and initiation of this clinical study are important steps forward in our mission to end the global organ shortage,” said Mike Curtis, President and Chief Executive Officer of eGenesis. “This trial will allow us to evaluate the transformative potential of EGEN-2784 to address the staggering burden of kidney failure worldwide.”
More than 800,000 Americans suffer from ESKD, however, only 28,000 kidney transplants were performed in 2024 due to organ shortage. The only alternative to transplant is dialysis, which has a five-year mortality rate of more than 50%. It's expected that pig kidney transplants will enable more people to receive this life-saving intervention. Right now, more than 100,000 people are on organ transplant waiting lists, and the majority are in need of a new kidney.
Meanwhile, researchers are also working on delivering genetically modified pig hearts to patients waiting for these organ transplants.
“Advances in xenotransplantation are giving our community hope that new options may soon be available to those who need them most,” said Kevin Longino, CEO of the National Kidney Foundation and a kidney transplant recipient. “The National Kidney Foundation congratulates eGenesis on this milestone. Patients expressed strong support for the advancement of clinical trials in our recent Xenotransplantation Voice of the Patient report. It is gratifying to know they’re being heard.”
Source: eGenesis
