Small molecule therapeutic for Steroid Resistant Nephrotic Syndrome (SRNS)

A compound for the potential treatment of SRNS. Developed by Professor Moin Saleem and colleagues at the University of Bristol in Bristol Renal.

Problem:

Steroid-resistant nephrotic syndrome (SRNS) is a devastating disease affecting both adults and children resulting from the breakdown of the kidney filtration barrier. It usually leads to end-stage renal disease (ESRD) despite the use of prolonged and toxic immunosuppression, with associated lifelong costs of dialysis and transplantation.

Solution:

Podocin is a key component of the glomerular filtration barrier and mutations in the podocin encoding gene NPHS2 lead to SRNS. These mutations or changes in genetic code represent the commonest form of inherited Nephrotic Syndrome (NS), up to 20% of all single-gene cases. The most frequent mutation is R138Q, causing mistrafficking of Podocin. We have tested a number of tool compounds that rescue this mistrafficking and restore normal function in our gold standard cell lines and a relevant and translatable mouse model.

Available Packages:

Bristol is seeking to establish partnerships with drug development companies in order to progress this therapeutic approach towards the clinic.

IP Status:

Patent pending – publication number WO2021/089692

Contact:

Email andrew.wilson@bristol.ac.uk for more information.

Photo by National Cancer Institute on Unsplash.

Other available technologies

See below for more technologies available technologies developed at the University of Bristol

Dr. Moin Saleem is a Professor at the University of Bristol and Director of Bristol Renal. He is founder and chief scientific officer of Purespring Therapeutics, the world's first renal gene therapy company.

Dr. Saleem's specific area is podocyte biology. His gene therapy program is focused on targeting the podocyte in order to radically change the treatment of kidney diseases.

Bristol Renal is an enthusiastic team of non-clinical and clinical scientists and students which aims to improve the management, treatment and prevention of renal disease by a strategy integrating laboratory and clinical research.

This study describes the generation and characterization of human kidney organoids representing an allelic series of homozygous NPHS2 missense variants.