ProQR Therapeutics N.V. (Nasdaq: PRQR) announced a global licensing and research collaboration with Eli Lilly and Company (Lilly) focused on the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The companies will use ProQR’s proprietary Axiomer® RNA editing platform to progress new drug targets toward clinical development and commercialization. ProQR’s unique Axiomer® platform technology enables the editing of single nucleotides in RNA in a highly targeted and specific manner. The technology is based on editing oligonucleotides, or EONs, designed to recruit endogenous ADAR enzymes (Adenosine Deaminases Acting on RNA) to a selected target adenosine in a disease associated RNA. ADAR then induces the conversion of the target adenosine (A) into inosine (I). The conversion from A to I is effectively an A to G change, as inosine in RNA is interpreted as a guanosine (G). This technology could be applied to potentially reverse the more than 20,000 G to A mutations in the human population that are known to cause disease.
For further information, see ProQR (https://www.globenewswire.com/news-release/2021/09/08/2293799/33039/en/ProQR-Announces-Axiomer-RNA-Editing-Licensing-and-Research-Collaboration-with-Lilly.html)
