Scientists at ProQR have invented a new way to use RNA oligonucleotides to alter RNA. The Axiomer® RNA editing technology enables the editing of specific single nucleotides in RNA. This technology could reverse the underlying cause of currently untreatable diseases.
Our Axiomer technology enables your body to repair its own RNA.
The first partner for the Axiomer® technology is Eli Lilly and Company. The global licensing and research collaboration focuses on the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The companies will use our proprietary Axiomer® RNA editing platform to progress up to five new drug targets toward clinical development and commercialization.
A new way to design oligonucleotide therapies
Antisense oligonucleotides (AONs) have been used as therapeutics for the last few decades. ProQR has built an extensive pipeline of investigational RNA therapies based on the technologies already available. But our scientists have gone beyond that and invented an entirely new way of using oligonucleotides for the treatment of genetic diseases. These Editing Oligonucleotides (EONs) make specific single nucleotide edits to RNA to reverse a mutation.
RNA base editing with ADAR
The ADAR (Adenosine Deaminase Acting on RNA) machinery is present in all human cells where it edits A’s to I’s in RNA. This A-to-I RNA editing is a very frequently occurring natural process. Our Axiomer platform evolved from the mechanism nature has developed.
The Axiomer technology
Our Axiomer EONs (Editing Oligonucleotides) can be designed to bind the mRNA at a specific site and mimics the double stranded RNA that normally attracts ADAR. This way the EON can induce editing of a specific ‘A’ to an ‘I’ where it is needed for therapeutic purposes. In short, we can design EONs to direct the cell to repair its own RNA with ADAR.
Applicability & potential
The EONs are short single stranded RNA molecules that are chemically modified to provide them with ideal drug-like properties for efficacy and uptake into cells. There are over 20,000 disease-causing mutations that can be ‘reversed’ by A-to-I editing. The vast majority of these diseases are currently untreatable.
Goals and strategy
We will further develop this platform through product and business development and plan to establish clinical proof of concept as soon as possible. The platform is uniquely positioned to target a wide range of diseases in a highly specific manner.
View this presentation for an overview of the Axiomer RNA editing technology opportunity.Internal link View presentation
Meet Bart Klein - Senior Vice President Innovation
"Axiomer opens up a whole new way of designing medicines"Internal link Read the interview