The 7th Annual Global Summit on Kidney Disease Innovations kicked off on May 23, 2025, with virtual sessions on rare disease, biomarkers, alternatives to human organs, and more. The event was hosted by the American Association of Kidney Patients (AAKP) and George Washington University (GW) School of Medicine and Health Sciences.
Rare Disease Updates
A session on breakthroughs for rare diseases began with Sharon McGroder, PhD, discussing autosomal dominant tubulointerstitial kidney disease (ADTKD). The inherited kidney disorders in this group often evade detection because they progress slowly and do not exhibit symptoms such as blood or protein in the urine, cysts, or a change in kidney size; thus, imaging and biopsy do not help with diagnosis.
Dr. McGroder quoted Anna Greka, MD, PhD, who has studied the molecular mechanisms underlying ADTKD, as saying, “Rare genetic diseases are collectively common.” Supporting that point, Dr. McGroder used ADTKD as an example of how research on rare diseases helps inform discovery for other diseases. She noted that insights on more than 50 disorders may be gained from ADTKD research, making investment in rare diseases critical.
Following Dr. McGroder’s segment, Daniel Jones, PhD, of Apellis discussed the role of complement activation in kidney, heart, ophthalmologic, and other diseases. Apellis developed Empaveli (pegcetacoplan), which was recently approved to treat paroxysmal nocturnal hemoglobinuria and is being investigated as a treatment for C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis, and other diseases.
David Fuller, BPharm, MBBS, of Dimerix discussed interim data from the ACTION3 study of the CCR2 inhibitor DMX-200 for focal segmental glomerulosclerosis, emphasizing the overall safety demonstrated so far. Addressing barriers to rare disease research, Dr. Fuller stated that patients are often unaware of trials, hindering participation. In addition, rare disease trials can be costly and inefficient because patients are spread out geographically, requiring multiple trial sites, he said.
Advancing Biomarkers
Another panel highlighted the Critical Path Institute’s (C-Path) Biomarker Data Repository (BmDR), which compiles large, reliable datasets on novel translational safety biomarkers from a variety of nonclinical and clinical study sources. This deidentified data will be used “to support research that leads to the submission of documents to global regulatory agencies to qualify novel safety biomarkers for new Contexts of Use (CoUs), to modify and expand existing CoUs, and to identify appropriate exploratory safety biomarkers to advance drug development in the future,” according to C-Path.
Katrina Peron of C-Path offered a demonstration of the BmDR, and Patrick Murray, MD, Joseph Bonventre, MD, and Stefan Sultana, MD, discussed the importance of advancing biomarkers. “We need better, more sensitive, and more specific tools,” Dr. Sultana said. A tool such as the BmDR can help kidney medicine mirror the successes of other specialties, advancing precision medicine and leading to better therapies and monitoring, Dr. Murray noted.
Human Kidney Alternatives
The day concluded with two sessions examining alternatives to human organ transplantation. The first focused on artificial implantable and wearable devices and 3D-printed kidneys.
Shuvo Roy, PhD, and William H. Fissell, MD, provided an overview of The Kidney Project, a national research effort to create a surgically implantable bioartificial kidney. Besides bypassing the problem of a donor shortage, such a device would also boast the advantage of not requiring immunosuppression, Dr. Roy pointed out. Xenotransplantation, on the other hand, requires immunosuppression, which can be problematic, and it is simply taking too long, Dr. Fissell said. At this point, Dr. Roy said, no fundamental discoveries are required to make the implantable device feasible; rather, it is an engineering effort.
Meanwhile, Kuleana, co-founded by Buddy Ratner, PhD, has been working to improve dialysis with more portable, convenient machines that continually refresh dialysate and require little water and no connection to an external water supply. The current prototype of AKTIV (Ambulatory Kidney to Increase Vitality) is about the size of a suitcase, but the next generation will be a wearable device, Dr. Ratner said. The company is developing a needleless vascular access technology, AKTIV Connection, to allow patients to connect multiple times a day without needlesticks and the risk of clots and infections.
Vidmantas Sakalys, CEO of Vital3D Technologies, closed out the session by talking about his company’s advances in 3D bioprinting. They are currently working on cell-laden structures, the second step on their journey to creating 3D organs. The reported progress is impressive. The company was founded in 2021 and expects to be creating organs by 2036.
The final session included transplant surgeon Robert Montgomery, MD, DPhil, who said that, whereas bioartificial organs and mechanical devices are still in development, xenotransplantation “has arrived.” He believes xenotransplantation may be approved within 5 years and on its way to scaling up.
He reviewed a few highlights of xenotransplantation’s history and the incredible progress in the field of late, which includes his own work performing the second transplantation of a gene-edited pig kidney to a living human at NYU Langone last year. That procedure used a 10-gene–edited porcine kidney, which will also be the focus of a human trial beginning with six participants and expanding to 50 over time.
Advancing Kidney Health Together
Day 2 of the summit featured sessions on vaccine efficacy, environmental and occupational exposures, progress in home dialysis, early intervention studies, APOL1 research, and insights on risk tolerance and dominant attribute.
These discussions not only highlighted the rapid pace of innovation in kidney care but also underscored the importance of collaboration among patients, healthcare professionals, researchers, private industry, and other stakeholders. For those who missed the event, sessions will be available on demand.
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