BMI OrganBank Reports the Publication of New Research Findings on Kidney Gene Therapy in Nature Biomedical Engineering

BMI OrganBank® is delighted to report the publication of new research findings in the prestigious journal Nature Biomedical Engineering – “Evolving adeno-associated viruses for gene transfer to the kidney via cross-species cycling of capsid libraries.” As part of this study, researchers from the laboratories of Dr. Aravind Asokan and Dr. Andrew Barbas at Duke University School of Medicine (Durham, NC, USA) performed the ex vivo machine perfusion of pig and non-human primate kidneys using an automated and portable perfusion system developed by BMI OrganBank®.

Problems related to the delivery of genetic material to the kidney have limited the translation of genetic medicines that could treat a significant proportion of the global population suffering from chronic kidney disease. Rosales et al. aimed to solve this problem by developing novel adeno-associated viruses (AAVs) as vectors that supported robust and widespread transduction of genetic material specifically to cells of the mouse, pig, and non-human-primate kidneys. While AAV vectors represent a U.S. Food and Drug Administration-approved technology for human gene therapies in ocular, hematological, and neuromuscular disorders, their application to kidney disease has been hampered by the intrinsic filtering function and complex physiology of the kidneys.

The authors of this fascinating new study engineered AAV variants with improved kidney transduction efficiency by implementing multiple distinct systems – intravenous administration in mice and pigs in vivo, infection of three-dimensional human kidney organoids (miniature cell-based models of organs) in vitro, and machine perfusion of isolated non-human primate kidneys ex vivo through BMI OrganBank® technology, OrganBank Lab – Pharmacodynamic Research Platform. The selected AAV variants transduced human kidney organoids and mouse/non-human primate kidneys with high efficiency and supported robust and widespread transduction when evaluated in pig kidneys preserved ex vivo via machine perfusion technology from BMI OrganBank®. Importantly, this latter finding highlights the translatability of these engineered AAV variants to the clinic, where they may be applied to kidney disease treatment and organ transplant applications in human patients.