eGenesis, a biotechnology company developing human-compatible engineered organs to address the global organ shortage, has announced clinical updates from an ongoing expanded access study of EGEN-2784, the company’s genetically engineered porcine kidney for the treatment of kidney failure.
Since March 2024, five patients with kidney failure have received EGEN-2784 kidney transplants under expanded access in collaboration with the Mass General Brigham health system (Boston, USA). Three of these patients have achieved sustained kidney function for more than eight months without the need for dialysis. One patient has surpassed nine months of dialysis independence, establishing a new global record for a genetically engineered porcine kidney transplant, and remains on study, according to the company. Two of the five patients have successfully transitioned to human donor kidney transplants.
These two cases represent the first reported transitions from xenotransplantation to allotransplantation and provide important clinical evidence that transplantation with a genetically engineered porcine kidney can support a subsequent transplant with a human donor kidney, the company states in a press release. The experience of one of these patients, Tim Andrews, was published in The Lancet. Andrews achieved approximately nine months of dialysis independence with an EGEN-2784 kidney before successfully receiving a human kidney 82 days following xenograft removal.
“These patient outcomes provide important evidence that genetically engineered organs may provide sustained kidney function and dialysis independence while preserving the opportunity for a subsequent human transplant,” said Mike Curtis, president and CEO, eGenesis. “Our goal is to make transplantation available to patients when they need it, without being constrained by the limited supply of human donor organs. Collectively, these experiences provide important clinical evidence as we prepare to advance EGEN-2784 into the phase 1/2/3 RESTORE study.”
The US Food and Drug Administration (FDA) has cleared eGenesis to conduct a phase 1/2/3 trial (RESTORE) to evaluate EGEN-2784 in 33 patients with kidney failure who are between 50 and 70 years of age and waitlisted for a human donor kidney transplant. eGenesis expects to initiate the RESTORE study in the first quarter of 2027.
EGEN-2784 is eGenesis’ lead candidate for kidney transplantation. The product carries three classes of genetic modifications designed to improve compatibility and support long-term function in human recipients: 1) elimination of three glycan antigens to prevent hyperacute immune rejection 2) insertion of seven human transgenes to regulate immune response, reduce inflammation, improve coagulation compatibility and regulate complement activation 3) inactivation of endogenous retroviruses to enhance safety.
Leonardo V Riella (Massachusetts General Hospital, Boston, USA) served as the principal investigator and led the clinical study, overseeing the multidisciplinary transplant team. Tatsuo Kawai was the lead transplant surgeon, supported by Nahel Elias, Massacusetts General Hospital’s surgical director for kidney transplantation.
“The experience with these patients is providing invaluable insight into the potential role of xenotransplantation for people living with kidney failure,” said Riella. “The sustained periods of dialysis independence are highly encouraging, and the successful transplantation of human donor kidneys following xenotransplantation demonstrates that this approach can preserve future treatment options. These findings provide an important foundation for the next stage of clinical development.”
