FDA Clears IND Application for EGEN-2784, a Genetically Engineered Pig Kidney, for ESKD

eGenesis has announced the US Food and Drug Administration (FDA) clearance of its Investigational New Drug (IND) application to initiate a clinical trial evaluating EGEN-2784, a genetically engineered porcine-derived kidney, in patients with end stage kidney disease (ESKD).1

According to a September 8, 2025, press release from the Company, the IND supports a phase 1/2/3 study designed to assess the safety, tolerability, and efficacy of EGEN-2784 at 24 weeks post-transplant in patients with ESKD who are ≥ 50 years of age, dialysis-dependent, and on the kidney transplant waitlist.1

“Advances in xenotransplantation are giving our community hope that new options may soon be available to those who need them most," Kevin Longino, CEO of the National Kidney Foundation and a kidney transplant recipient, said in a statement.1 “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 eGenesis’ lead candidate for kidney transplantation and carries 3 classes of genetic modifications designed to improve compatibility and support long-term function in human recipients:

• Elimination of 3 glycan antigens to prevent hyperacute immune rejection
• Insertion of 7 human transgenes to regulate immune response, reduce inflammation, improve coagulation compatibility and regulate complement activation
• Inactivation of endogenous retroviruses within the porcine genome to enhance safety

Of note, eGenesis is the only company developing organs with all 3 classes of edits to optimally address safety and efficacy.1

“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, PhD, President and Chief Executive Officer of eGenesis.1 “This trial will allow us to evaluate the transformative potential of EGEN-2784 to address the staggering burden of kidney failure worldwide.”

As described in the release from eGenesis, on January 25, 2025, Tim Andrews, 67, received an EGEN-2784 kidney at Massachusetts General Hospital in the institution’s second successful transplant of a genetically edited pig kidney into a living recipient.2 He has surpassed 7 months post-transplant, making him the world’s longest-living recipient of a genetically engineered porcine-derived organ. After previously requiring dialysis for > 2 years before his transplant, Andrews continues to live dialysis-free.1

On June 14, 2025, Bill Stewart, 54, became the second patient in the study, and the third patient overall, to receive an EGEN-2784 kidney. Like Andrews, Stewart no longer requires dialysis for the first time in > 2 years.1

“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,” Stewart said.1

Leonardo Riella, MD, PhD, medical director for kidney transplantation at Massachusetts General Hospital, served as the principal investigator and led the clinical study, overseeing the multidisciplinary transplant team. Tatsuo Kawai, MD, PhD, director of the Legorreta Center for Clinical Transplant Tolerance, was the lead transplant surgeon for both procedures, supported by Nahel Elias, MD, surgical director for Kidney Transplantation.1

“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,” said Leonardo Riella, MD, PhD, medical director for kidney transplantation at Massachusetts General Hospital and principal investigator of the clinical study.1 “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.”

References

1. eGenesis. eGenesis Announces IND Clearance for EGEN-2784 in Kidney Transplant and Landmark Patient Updates in Ongoing Expanded Access Study. September 8, 2025. Accessed September 8, 2025. https://egenesisbio.com/press-releases/egenesis-announces-ind-clearance-for-egen-2784-in-kidney-transplant-and-landmark-patient-updates-in-ongoing-expanded-access-study/

2. Brooks A. Second Xenotransplant with Gene-Edited Pig Kidney Performed at Mass General. HCPLive. February 7, 2025. Accessed September 8, 2025. https://www.hcplive.com/view/mass-general-performs-second-xenotransplant-gene-edited-pig-kidney