eGenesis Announces Clinical Update in Ongoing Expanded Access Study of EGEN-2784 in Patients with Kidney Failure

Five patients have received EGEN-2784 kidney transplants under Expanded Access since March 2024

FDA-cleared Phase 1/2/3 clinical trial of EGEN-2784 expected to initiate in 1Q 2027

CAMBRIDGE, Mass.--(BUSINESS WIRE)--eGenesis, a biotechnology company developing human-compatible engineered organs to address the global organ shortage, announced today 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. 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. 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 experience of one of these patients, Tim Andrews, was published today 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, Ph.D., President and Chief Executive Officer, 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 U.S. 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, M.D., Ph.D., Medical Director for Kidney Transplantation at MGH, served as the principal investigator and led the clinical study, overseeing the multidisciplinary transplant team. Tatsuo Kawai, M.D., Ph.D., Director of the Legorreta Center for Clinical Transplant Tolerance, was the lead transplant surgeon, supported by Nahel Elias, M.D., 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 Dr. 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.”

More than 800,000 Americans and millions worldwide suffer from end-stage kidney disease (ESKD). While organ transplantation remains the gold standard for improving survival and quality of life, the severe shortage of donor organs limits access to transplantation. Only 28,000 kidney transplants were performed in the U.S. in 2024. For those unable to receive a transplant, dialysis remains the only option. The five-year mortality rate associated with dialysis is 60%, higher than mortality rates associated with the most common cancers. Genetically engineered porcine-derived kidneys offer a promising solution to this crisis.

About eGenesis

eGenesis is a clinical-stage biotechnology company developing human-compatible engineered organs to address the severe global organ shortage. The Company’s proprietary genome engineering platform enables multiplex gene editing designed to address key barriers to xenotransplantation, including immune rejection and pathogen safety. EGEN-2784, a genetically engineered porcine kidney, is the Company’s lead program. eGenesis is also developing products for liver and heart failure. To date, the Company has received FDA clearance to conduct clinical studies across its kidney and liver programs. eGenesis is headquartered in Cambridge, MA. For more information, visit www.egenesisbio.com.