Since 2021 ProQR has had a global licensing and research collaboration with Eli Lilly and Company. The partnership focuses on the discovery, development, and commercialization of potential new medicines for genetic disorders.
The ongoing collaboration uses ProQR’s proprietary Axiomer™ RNA editing platform to progress multiple RNA editing drug targets toward clinical development and commercialization.
Discovering and developing the medicines of tomorrow takes time, sustained innovation and most importantly, collaboration.
“Discovering and developing the medicines of tomorrow takes time, sustained innovation and most importantly, collaboration. We have been impressed with the progress to date with our partners at ProQR and have conviction that RNA editing can be an important alternative to other more permanent therapies,” says Andrew C. Adams, Ph.D., Lilly senior vice president of genetic medicine and co-director of the Institute for Genetic Medicine.
Since ProQR was founded, the company has focused on RNA technologies to develop medicines for people and families with high unmet medical need. Through the course of work with Lilly, advances in the platform have significantly increased editing efficiency and refined biodistribution in both the liver and nervous system, opening new potential applications to not only correct known mutations, but also introduce protective variants in specific transcripts.
Through its ongoing collaboration, Lilly and ProQR will explore further applications to unlock new innovative treatments for people living with diseases with high unmet medical need.
ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer™ “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer™ EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.
“Our original collaboration with Lilly, which leverages our Axiomer™ RNA editing technology platform, continues to progress well and we are pleased to be expanding our partnership to include additional targets, along with an option for Lilly to opt in for more. Lilly is a leader in RNA therapeutics, and our expanded partnership is another validation of our leadership in ADAR-mediated RNA editing, our robust IP estate, and the potential of our broadly applicable Axiomer™ platform technology. We look forward to making an impact on the lives of patients together with Lilly,” says Daniel A. de Boer, founder, and CEO of ProQR.
For more information, please read our collaboration press releases:
Since January 2024 ProQR has had a collaboration with the Rett Syndrome Research Trust (RSRT) that will focus on the design and development of editing oligonucleotides (EONs) using ProQR’s Axiomer technology platform targeting the transcription factor Methyl CpG binding protein 2 (MECP2) and correcting mutations of interest. The partnership was expanded in December 2024.