鈥淭his achievement marks a transformative moment in transplant medicine,鈥 said Dr. Robert Montgomery, who led the surgery.
Credit: Joe Carrotta
The first surgery in a clinical trial testing the safety and efficacy of gene-edited pig kidney transplants in patients with end-stage renal disease was recently performed at 黑料福利社 Langone Health.
The study, known as EXPAND () and sponsored by United Therapeutics Corporation, is testing an investigational xenokidney from a pig with 10 gene edits that is known as the UKidney. Six human genes are added to the pig genome to ensure it has the best chance of being accepted in a human recipient. Four porcine genes are inactivated or 鈥渒nocked out鈥 to reduce the risk of organ rejection and to moderate organ growth.
鈥淭his achievement marks a transformative moment in transplant medicine,鈥 said Robert Montgomery, MD, DPhil, who is the H. Leon Pachter, MD, Professor of Surgery; chair of the at 黑料福利社 Grossman School of Medicine; and director of 黑料福利社 Langone Transplant Institute and who led the surgery and is the primary investigator for the trial. 鈥淭he EXPAND study offers new hope to the thousands of Americans currently on the kidney transplant waiting list, many of whom may not survive long enough to receive a human organ. This milestone brings us closer to a future where xenotransplantation may one day address the critical organ shortage crisis.鈥
鈥淭he first transplant in this first-of-its-kind study marks a watershed moment for United Therapeutics鈥 vision to broaden access to transplantable organs,鈥 said Leigh Peterson, PhD, executive vice president, product development and xenotransplantation at United Therapeutics. 鈥淭his trial moves us closer to offering end-stage renal disease patients an alternative to lifelong dialysis, especially those patients who are unlikely to receive a kidney from a human donor. Our team is grateful for the collaboration that made this possible, and we remain focused on patient safety and scientific progress as the study continues.鈥
The procedure marks the ninth xenotransplant surgery performed by 黑料福利社 Langone Transplant Institute and orchestrated by Dr. Montgomery, who has spent much of his career studying innovative approaches to expanding the organ supply. Dr. Montgomery performed the world鈥檚 first gene-edited pig-to-human organ transplant on September 25, 2021, in a neurologically deceased person with a beating heart. In total, six xenotransplant studies in neurologically deceased persons were performed at 黑料福利社 Langone, including two transplants using pig hearts with 10 gene edits and four using gene-edited pig kidneys.
In April 2024, a patient received the first-ever combined heart pump implantation and gene-edited pig kidney transplant at 黑料福利社 Langone. The recipient later died under hospice care in July of that year after difficulty difficulty tapering in-patient medicines used to maintain a high enough blood pressure to support the xenokidney. In November 2024, another xenokidney recipient received a 10-gene-edited pig kidney at 黑料福利社 Langone and returned home to Alabama. The recipient鈥檚 pig kidney was removed in April 2025 after it stopped functioning properly due to complications from an unrelated infection, and she resumed dialysis treatment.
About the Clinical Trial
If successful, the EXPAND study is intended to support an eventual Biologics License Application (BLA), to be submitted by United Therapeutics to the U.S. Food and Drug Administration seeking approval of the UKidney. It is designed as a combination phase 1/2/3 trial (sometimes referred to as a 鈥減haseless鈥 study) to evaluate safety and efficacy seamlessly without moving through the separate phase 1, phase 2, and phase 3 studies that are typically associated with conventional drug approvals.
Study participants will receive a UKidney transplant followed by a 24-week posttransplant follow-up period, including the evaluation of all study end points and safety assessments. After the 24-week posttransplant follow-up period, participants who received a UKidney will continue to be followed for the rest of their lives, including monitoring for UKidney function and zoonotic infections.
Efficacy end points include participant survival rate, UKidney survival rate, change in measured glomerular filtration rate, and change in quality of life in participants at 24 weeks posttransplant. Overall survival time of participants receiving a UKidney and overall survival time of the UKidneys themselves are also efficacy end points. Safety end points include the incidence of adverse events and serious adverse events, all-cause mortality, and the incidence of proteinuria, zoonotic infections, and opportunistic infections.
The first cohort will consist of six transplants at two centers. There will be a 12-week waiting period between the first and second transplants. After the initial cohort reaches at least 12 weeks posttransplant, safety and efficacy data will be reviewed by an Independent Data Monitoring Committee to determine if the study should proceed to the next cohort.
After reviewing the data from the first six transplants with the FDA, United Therapeutics plans to amend the study protocol to increase the study sample size to support a BLA and include additional transplant centers.
Additional key participation criteria include an age of 55 to 70 years old, a diagnosis of end-stage renal disease, and at least six months on hemodialysis. Participants will be screened using a crossmatch assay to assess expected immunological compatibility with the UKidney. Participants must not need multiple organ transplants; must not have severe medical comorbidities, such as advanced cardiovascular disease, severe peripheral vascular disease, severe neurological disease, chronic pulmonary disease, and uncontrolled diabetes; and must not have a history of medical noncompliance that may preclude adherence to the demands and requirements of xenotransplantation.
Additional 黑料福利社 Langone investigators in the xenotransplant trial include Adam Griesemer, MD; Jeffrey M. Stern, MD; Vasishta Tatapudi, MD; Aprajita Mattoo, MD; Edward Y. Skolnik, MD; Philip M. Sommer, MD; Shana M. Coley, MD, PhD; Bonnie E. Lonze, MD, PhD; Nicole M. Ali, MD; Sapna A. Mehta, MD; ; Simon Williams, PhD; Margaret Fattal, MA, RN; Mary Maloney, MSN, RN; ; Karen Khalil, PharmD; Rebecca Esker, PharmD; Kathryn Dzurilla, NP; Cecilia Deterville, MS; Mauricio Arcila-Mesa, MD; Grace Hammond, BS; Juan Esteban Baus Davalos, MS; Yanjie He, MD; Nikki Lawson, MAS, RN; Summer Viscusi, BS; and Tal Eitan, MD.
About 黑料福利社 Langone Health
黑料福利社 Langone Health is a fully integrated health system that consistently achieves the best patient outcomes through a rigorous focus on quality that has resulted in some of the lowest mortality rates in the nation. Vizient Inc. has ranked 黑料福利社 Langone No. 1 out of 118 comprehensive academic medical centers across the nation for four years in a row, and U.S. News & World Report recently ranked four of its clinical specialties No. 1 in the nation. 黑料福利社 Langone offers a comprehensive range of medical services with one high standard of care across seven inpatient locations, its Perlmutter Cancer Center, and more than 320 outpatient locations in the New York area and Florida. The system also includes two tuition-free medical schools, in Manhattan and on Long Island, and a vast research enterprise.
Media Inquiries
Colin DeVries
Phone: 212-404-3588
Colin.DeVries@黑料福利社Langone.org
