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Chronic kidney failure

First Clinical Trial Evaluates Xenotransplantation in End-Stage Renal Disease

Strategic Insights

— 2 min read

In a significant breakthrough in the field of transplantation, the EXPAND study has achieved a major milestone with the first clinical xenotransplantation in patients with end-stage renal disease (ESRD). Performed at NYU Langone Health, the successful transplant operation utilized an investigational xenokidney from a pig with 10 gene edits, designed to facilitate immunological acceptance and compatibility in the human recipient. This achievement marks a transformative moment in transplant medicine, offering new hope to the thousands of Americans on the kidney transplant waiting list.

Key Takeaways:

  • The EXPAND study is a multicenter, open-label, safety and efficacy study designed to evaluate the safety and efficacy of the UKidney xenokidney in patients with ESRD.
  • The study aims to support a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) and will involve a combination phase 1/2/3 trial to evaluate safety and efficacy seamlessly without moving through separate phase 1, phase 2, and phase 3 studies.
  • Study participants will receive a UKidney transplant followed by a 24-week post-transplant follow-up period, including the evaluation of all study endpoints and safety assessments.
  • The UKidney xenokidney has 10 gene edits, including the addition of six human genes to facilitate immunological acceptance and the inactivation of four porcine genes to reduce the risk of organ rejection and moderate organ growth.
  • The study will include six transplants at two centers, with a 12-week waiting period between the first and second transplants, and will be reviewed by an Independent Data Monitoring Committee to determine if the study should proceed to the next cohort.
  • Additional key participation criteria include an age of 55 to 70 years old, a diagnosis of ESDR, and at least six months on hemodialysis, as well as screening using a crossmatch assay to assess expected immunological compatibility with the UKidney.

Statistics:

  • Approximately 815,000 patients have kidney failure in the United States, and more than 555,000 patients are on dialysis, with approximately 90,000 patients on the U.S. kidney transplant waiting list.
  • Only 22,000 deceased donor kidney transplants occurred in 2024, and a 2021 study found that three years after starting dialysis, only 12% of patients had been placed on an Organ Procurement and Transplant Network kidney transplant waitlist while more than 40% died.

Sources:

  • United Therapeutics Corporation press release, "United Therapeutics Announces First Clinical Xenotransplantation in EXPAND Study" (November 3, 2025)
  • American Kidney Fund, "All About Kidneys: Quick Kidney Disease Facts and Stats" (2025)
  • US Renal Data System, "2023 End-Stage Renal Disease: 7. Transplantation" (2023)

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