eGenesis, a biotechnology firm focused on developing human-compatible engineered organs, announced that the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application to begin a clinical trial for EGEN-2784, a genetically engineered porcine-derived kidney aimed at treating patients with end-stage kidney disease (ESKD).

The IND approval facilitates a Phase 1/2/3 study to evaluate the safety, tolerability, and effectiveness of EGEN-2784 at 24 weeks after transplantation in patients aged 50 and older who are dependent on dialysis and on the kidney transplant waitlist.

“Clearance of the EGEN-2784 IND and initiation of this clinical study are important steps forward in our mission to end the global organ shortage,” said Mike Curtis, Ph.D., President and Chief Executive Officer of eGenesis. “This trial will allow us to evaluate the transformative potential of EGEN-2784 to address the staggering burden of kidney failure worldwide.”

Kevin Longino, CEO of the National Kidney Foundation and a kidney transplant recipient, expressed optimism regarding advancements in xenotransplantation: “Advances in xenotransplantation are giving our community hope that new options may soon be available to those who need them most. The National Kidney Foundation congratulates eGenesis on this milestone. Patients expressed strong support for the advancement of clinical trials in our recent Xenotransplantation Voice of the Patient report. It is gratifying to know they're being heard.”

EGEN-2784 is the leading candidate from eGenesis for kidney transplantation. The kidney features three genetic modifications aimed at enhancing compatibility and promoting long-term function: 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 manage complement activation, and 3) Inactivation of endogenous retroviruses within the porcine genome to improve safety. eGenesis stands out as the only company implementing all three classes of edits to optimally ensure safety and efficacy.

Major milestones have been reached in the ongoing Multi-Patient Expanded Access study of EGEN-2784. Tim Andrews, 67, received an EGEN-2784 kidney on January 25, 2025, at Massachusetts General Hospital. He has surpassed seven months post-transplant, making him the world’s longest-living recipient of a genetically engineered porcine-derived organ. Mr. Andrews, who was on dialysis for over two years before his transplant, is now living without dialysis.

On June 14, 2025, Bill Stewart, 54, became the second participant in the study and the third overall to receive an EGEN-2784 kidney. Mr. Stewart had been undergoing dialysis three times weekly for over two years and had faced a prolonged wait for an allotransplant due to his O-group blood type. He was discharged from MGH on June 21st and is no longer reliant on dialysis for the first time in over two years.

“I’m so appreciative of the MGH transplant team for giving me this opportunity and a new lease on life. The care, compassion, kindness, support, and intelligence that the team has invested in my care and studying xenotransplantation will forever be appreciated by my family and me. The commitment to my care has been evident from day one, and it has allowed me to trust in the process and research. I couldn’t be in better hands,” Mr. Stewart expressed.

Leonardo V. Riella, MD, PhD, the medical director for kidney transplantation at MGH, led the clinical study as Principal Investigator, overseeing the multidisciplinary transplant team. Tatsuo Kawai, MD, PhD, who serves as the Director of the Legorreta Center for Clinical Transplant Tolerance, was the lead surgeon for both transplant procedures, with support from Nahel Elias, MD, Surgical Director for Kidney Transplantation.

“This is a historic moment for xenotransplantation. To see a patient thriving off dialysis for over seven months with a genetically engineered pig kidney is nothing short of groundbreaking,” remarked Dr. Riella. “This achievement was made possible by extraordinary collaboration across academia, industry, and government. I want to recognize eGenesis for its cutting-edge gene-editing technology and essential partnership; our MGH transplant team for their clinical excellence; and the FDA for their rigorous and supportive guidance. Additional critical industry partners include Eledon, Apellis, and Karius. Together, we are not only making history—we are reshaping the future of transplantation.”

With more than 800,000 Americans and millions globally suffering from ESKD, organ transplantation remains the best option for improving survival and quality of life. However, only 28,000 kidney transplants were performed in 2024 due to the organ shortage. For those unable to receive a transplant, dialysis remains the sole alternative, with a five-year mortality rate exceeding 50%, higher than that of the most common cancers. Genetically engineered porcine-derived kidneys present a promising solution to this urgent crisis.

eGenesis, based in Cambridge, MA, is a clinical-stage biotechnology company focused on creating human-compatible engineered organs to alleviate the global organ shortage. Their proprietary genome engineering platform allows for extensive gene edits to remove biological barriers, introduce protective human transgenes, and inactivate endogenous retroviruses. EGEN-2784 is currently being evaluated in a multi-patient Expanded Access study at MGH.

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