The National Kidney Foundation (NKF) today announced an investment from the NKF Innovation Fund in Nephrogen, a biotechnology company developing one-time gene editing therapies for kidney disease.
Gene therapy has opened new possibilities for treating and potentially curing diseases by addressing their underlying genetic causes. But bringing those advances to kidney disease has proven particularly challenging. According to the Q2 2026 landscape report from the American Society of Gene and Cell Therapy (ASGCT), 931 gene therapies are approved, in clinical trials, or awaiting regulatory approval worldwide. No gene therapy has been approved for a kidney disease, and only one program designed to target kidney cells is known to have entered clinical trials. The kidney’s complex structure and filtration barrier make it difficult for gene therapy delivery vehicles to reach their target cells, and kidney disease can originate in more than 15 distinct cell types.
Nephrogen’s NeFIND™ platform, which combines artificial intelligence with high-throughput screening to discover new gene therapy delivery vehicles, aims to address these challenges to make gene therapy a reality for the 37 million Americans living with kidney disease. In work currently under peer review, Nephrogen reported delivery to as much as 60% of kidney tubular epithelial cells in preclinical mouse models. Nephrogen has also demonstrated gene delivery in intact human kidneys taken directly from patients and maintained on normothermic machine perfusion which to the company’s knowledge is the first reported demonstration of targeted delivery in intact human kidney tissue.
“Gene therapy is changing what is possible for patients with diseases that once had few or no treatment options, however, no gene therapies are currently available to treat kidney diseases,” said Kevin Longino, Chief Investment Officer of NKF and a kidney transplant recipient. “Nephrogen is tackling a fundamental challenge that has stood in the way: getting these potentially transformative therapies into the kidney. This investment reflects exactly what the NKF Innovation Fund was created to do: help accelerate promising innovations with the potential to save lives and fundamentally change the future of kidney disease.”
Nephrogen’s lead program is a one-time gene editing therapy for autosomal dominant polycystic kidney disease (ADPKD), the most common inherited kidney disease. A second program targets APOL1-mediated kidney disease and Nephrogen’s longer-term goal is to apply the NeFIND™ platform to a much broader range of kidney diseases, including chronic kidney disease.
“Families with inherited kidney disease have spent decades being told to manage decline rather than treat its cause,” said Demetri Maxim, CEO and co-founder of Nephrogen. “That isn’t because the genetics are unknown, it’s because delivering genetic material into the specific kidney cell types that drive the disease has been far too inefficient. We built our NeFIND™ platform to change that and so we can make one-time gene editing therapies a reality for patients who have been waiting for far too long. Polycystic kidney disease runs in my own family, and that’s what makes this especially urgent for me.”
The NKF Innovation Fund investment will support expansion of the NeFIND™ platform to enable efficient delivery to additional kidney cell types and advance Nephrogen’s lead ADPKD program toward IND-enabling studies.
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