Drug intelligence / Profile preview

UKidney

Development stage
Phase 2
Lead developer
United Therapeutics
Modality
Tissue Cells → Other Cell Types → Cell Therapies
Administration
Surgical Transplantation
01

Overview

UKidney is a genetically engineered porcine kidney developed by United Therapeutics, through its subsidiary Revivicor, for xenotransplantation in patients with end-stage renal disease (ESRD). The organ features 10 specific genetic modifications (10 GE) designed to overcome the immunological barriers of pig-to-human transplantation. These edits include the knockout of three porcine carbohydrate antigens (GGTA1, CMAH, and B4GALNT2) to prevent hyperacute rejection, the knockout of the porcine growth hormone receptor (GHR) to prevent organ overgrowth, and the expression of six human transgenes. These transgenes include complement regulators (CD46, CD55, and CD59), anticoagulants (thrombomodulin and endothelial protein C receptor), and an immunomodulator (CD47). UKidney is currently being evaluated in the EXPAND clinical trial for ESRD patients who are ineligible for or unlikely to receive a human kidney transplant.

Brand names
UKidney
Other names
Genetically engineered porcine kidney10-gene edited pig kidney
02

Targets

CD47 (Cluster of Differentiation 47)Cytidine monophosphate-N-acetylneuraminic acid hydroxylase / N-glycolylneuraminic acid xenoantigen pathwayGHR (Growth hormone receptor)THBD (Thrombomodulin)SIRPA (Signal-regulatory protein alpha)CD46 (CD46 Molecule)HMOX1 (Heme oxygenase 1)PROC (Protein C)EPCR (Endothelial protein C receptor)CD55 (Complement decay-accelerating factor)B4GALNT2 (Beta-1,4-N-acetyl-galactosaminyltransferase 2)

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