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2025-02-04| In-DepthTrending

A Win for Transplant Medicine — FDA IND Approval for Gene-Edited Pig Kidney Clinical Trial

by Denisse Sandoval
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United Therapeutics Corporation has announced the launch of the first-ever human clinical trial of a xenotransplantation intended to support potential registration through a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA). The FDA has approved the company’s Investigational New Drug (IND) application to begin the study. The trial will evaluate UKidney, a pig-derived organ with 10 gene edits. The trial will begin with six patients with end-stage renal disease. It may later expand to include up to 50 participants. United Therapeutics expects the first transplant in the trial to take place by mid-2025.

UKidney Trial Eligibility Requires Ages 55-70, Six Months of Hemodialysis, and Immunological Screening

This first-in-human clinical trial openly evaluates UKidney’s safety and efficacy. The trial uses a combined phase 1/2/3 approach, enabling continuous evaluation of safety and efficacy without separate trial phases. Participants will undergo a UKidney transplant and a 24-week post-transplant follow-up to assess study endpoints and safety. Patients who receive a UKidney will undergo lifelong monitoring to assess survival, organ function, and the risk of zoonotic infections.

The clinical trial will enroll and evaluate two groups of patients with ESRD. The first group includes those deemed medically ineligible for a conventional allogeneic kidney transplant. Furthermore, the second group includes waitlisted patients with a higher risk of dying. They are unlikely to receive a donor kidney within five years.

Eligible participants for the trial must have undergone at least six months of hemodialysis. The study will use a crossmatch assay to assess immunological compatibility with the UKidney. Additionally, candidates cannot require multiple organ transplants. They must not have severe co-morbidities like cardiovascular, neurological, or pulmonary disease. The study will exclude individuals with a history of medical noncompliance that could impact adherence to xenotransplantation requirements.

Leigh Peterson, Ph.D., Executive Vice President, Product Development and Xenotransplantation at United Therapeutics, stated, “Our goal is to increase the availability of transplantable organs to offer a therapeutic alternative to a lifetime on dialysis for a large population of patients who are unlikely to receive an allogeneic kidney transplant.”

Only 12% of Dialysis Patients Make the Transplant Waitlist, While Over 40% Die

The American Kidney Fund reports that approximately 808,000 people in the United States have kidney failure, with over 557,000 relying on dialysis. A 2009 study estimated that 52% of kidney transplant candidates aged 60 or older die within five years of being placed on the waitlist before receiving a transplant. 

Around 93,000 patients are on the national kidney transplant waiting list, yet only 21,000 deceased donor kidney transplants were performed in 2023. A 2021 study found that within three years of starting dialysis, just 12% of patients were placed on the Organ Procurement and Transplant Network waitlist, while more than 40% died. 

United Therapeutics considers xenotransplantation a potential solution for patients dependent on dialysis. Its investigational organ, UKidney, is derived from a pig with 10 gene edits designed to improve compatibility with the human immune system. United Therapeutics adds six human genes to enhance organ acceptance. Additionally, it also inactivates four porcine genes—three to reduce rejection risk and one to control growth.. This approach aims to address the critical shortage of donor kidneys and provide a long-term treatment option for ESRD patients.

“Eliminating the need for dialysis or limiting time on dialysis may improve survival for many patients with ESRD,” said Noah Byrd, Ph.D., RAC, Vice President, Global Regulatory Affairs at United Therapeutics. “We appreciate the productive collaboration with the FDA in advancing our efforts to bring this potentially revolutionary therapeutic option to the hundreds of thousands of ESRD patients.”

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