OUR LEAD FORMULATION
A short summary of the problem and why we are so determined to solve it
Chronic kidney disease
A serious problem
More than 850 million people in the world have chronic kidney disease (CKD), a number that is still on the rise. CKD is a collective term for a wide variety of diseases with progressive loss of kidney function, ultimately leading to chronic kidney failure.
In many of these diseases, damage is done to the glomerulus, the filtration unit of the kidney. In response to this damage, inflammation is initiated, which often causes more damage, constituting a vicious cycle, ultimately leading to complete kidney failure.
Once insufficient kidney function is left, a condition referred to as end-stage kidney disease, individuals are dependent on kidney replacement therapy (dialysis or kidney transplantation).
Current treatment of CKD
“A zero sum game”
The underlying conditions that cause the initial damage and start the vicious cycle leading to kidney failure include diabetes, hypertension, and/or auto-immune diseases.
Despite the differences in aetiology, glomerular inflammation is the driving factor behind progression in each of these diseases. Therefore, the major component of CKD treatment consists of immune-suppressive drugs, and has been for the last 50 years.
The systemic nature of these drugs is the reason for the frequently reported side-effects including fatigue, infections, infertility and even cancer. These side-effects are often so severe that they become dose-limiting, thereby leading to insufficient treatment of the disease. As a result, patients often still progress towards end-stage kidney disease and associated kidney replacement therapy. Thus, there is a great need for new medication for CKD.
Precision medicine for CKD
At Mercurna, we are looking into all kinds of aspects related to the individual components, combinations of components, pathways to target, etc.
Our lead formulation consists of an unique cocktail of mRNAs, encoding for therapeutic proteins, that attacks the disease at its root and brings back the homeostasis in the kidney. These mRNAs are encapsulated in lipid nanoparticles that are labeled with our targeting peptide, in order to deliver the mRNAs straight into the glomerulus, without interfering with the rest of the body in order to prevent side-effects.
The unprecedented targeting and powerful activity makes this a true precision medicine, which we believe is applicable to a wide variety of CKD indications.