Targeted Delivery of siRNA as a Novel Therapy to Treat Acute Kidney Injury

SK2013-058 and SK1175

Targeted Delivery of siRNA as a Novel Therapy to Treat Acute Kidney Injury

SK2013-058 and SK1175


Acute kidney injury (AKI), characterized by a sudden loss of kidney function within 48 hours, is a frequent consequence of standard-of-care medications and surgical procedures that unavoidably produce nephrotoxic or ischemic damage. AKI is reported among 2-5% of patients during hospitalization, and is associated with very high rates of mortality (greater than 50% among ICU patients). The current standard of care for AKI patients is largely supportive—after injury is diagnosed, primary treatment goals focus on maintaining volume homeostasis and correcting biochemical abnormalities. Currently, there is no FDA approved drug to either prevent or treat AKI.

MSK investigators have developed a novel therapeutic strategy to treat or prevent AKI. This potentially transformative technology utilizes fibrillar particles to deliver therapeutic doses of small interfering RNA (siRNA) to renal proximal tubules, where they inhibit signaling pathways associated with renal damage. In mouse models of cisplatin-induced and ischemia/reperfusion-induced kidney injury, RNA interference from the MSK agent prevents both nephrotoxic and ischemic kidney injuries. The fibrillar particle platform mediates the tissue- and cell-specific delivery of siRNA, which is biocompatible and superior to administration of RNA alone. This drug is similarly bioactive in human renal cells in vitro under nephrotoxic or ischemic conditions.


  • Initial in vivo studies suggest favorable biocompatibility, tissue distribution, and clearance, with proof-of-concept demonstrated in mouse models of kidney injuries
  • Safety has been demonstrated in non-human primates and rodents at pharmacological doses
  • Potential first-in-class therapeutic treatment option for AKI patients


According to the American Society of Nephrology, there are 1.2 million patients who suffer from AKI per year in the U.S.  Approximately 30-40% of patients who receive cisplatin treatment acquire AKI, requiring suspension of cisplatin treatment and switching to an alternative chemotherapy. With an estimated 13.3 million cases and 1.7 million deaths worldwide each year, there is a strong unmet need for a strategy to treat or prevent AKI.


  • Alidori S. et al., Targeted Fibrillar Nanocarbon RNAi treatment of acute kidney injury. Science Translational Medicine, 8(331), 2016 (PubMed ID: 27009268)
  • Ruggiero A. et al., Paradoxical glomerular filtration of carbon nanotubes. Proceedings of the National Academy of Science, USA, 107(27), 2010 (PubMed ID: 20566862)




PCT application 2016/014808 published January 2016 and U.S. Patent 8,540,965 issued September 24, 2013


  • Michael R. McDevitt, PhD, ME, Member, Sloan Kettering Institute; Attending, Memorial Hospital, Center for Molecular Imaging & Nanotechnology, and the Brain Tumor Center, MSK
  • David A. Scheinberg, MD, PhD, Program Chair, Molecular Pharmacology Program, Sloan Kettering Institute, and Attending Oncologist, Department of Medicine, Memorial Hospital, MSK


James Delorme, PhD, Licensing Manager

Tel # 332-229-0588
E-mail: [email protected]

Stage of Development

In vitro