Public Funding for Ninevah Therapeutics Ltd

58,000 individuals with end-stage kidney disease in the UK are at risk of dying from kidney failure. There are not enough donor kidneys, transplanted organs often only last a few years, and dialysis strongly disrupts patient's lives and is very expensive to the economy. Severe kidney diseases are caused by single gene mutations. Alport Syndrome is a relatively common inherited kidney disease that affects 10k patients in the UK. Risk factors like obesity, diabetes, and hypertension can accelerate such conditions and are on the increase, underlining the need for new curative treatments. Current and emerging medicines can buy Alport Syndrome patients some time, but they do not cure the underlying cause of the disease. By contrast, gene therapy offers a potential cure by replacing the mutated gene. However, this is especially challenging in the kidney due to the nature of its blood flow and connection with the rest of the body. Few kidney gene therapy platforms are in development, and those that are show limited cell-specific control, tissue-targeted delivery, and cargo gene capacity. As a result, only a small percentage of the many identified inherited kidney diseases are targetable, and any potential future gene therapy product bears with it unnecessary risks. Ninevah Therapeutics have developed a new kidney gene therapy vector with superior control of cargo gene expression, more space to fit large genes and therefore target more rare kidney disorders, and a roadmap for tissue-specific delivery to make the treatment as safe and widely available as possible. With the University of Manchester's Prof Rachel Lennon, a globally renowned expert in Alport Syndrome, Ninevah will test the new medicine as a first proof-of-principle for tackling rare inherited kidney diseases. The project propels the research effort towards human trials and future introduction of this breakthrough medicine for patients. There is potential to secure health economic savings amounting to £2.5Bn within 5 years of first reaching patients. Ninevah's gene therapy platform is potentially relevant to over 450 known MKD mutations, collectively responsible for around 12% of all end-stage kidney disease. In addition, due to similarities in the type of gene that is mutated, the testing strategy for Alport Syndrome may also help scientists understand a range of other rare diseases unrelated to the kidney, such as bleeding in the brain, brittle bone disease and muscle wasting disorders.