ProQR Therapeutics N.V. has 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.

”RNA editing is an exciting emerging technology, which allows transient, reversible editing, which in some indications may be an extremely attractive therapeutic approach” said Andrew C. Adams, Ph.D., vice president for New Therapeutic Modalities at Lilly. “Through this collaboration with ProQR, we hope to utilize this technology to unlock novel treatments to improve the lives of patients across a spectrum of diseases.”

“This partnership with Lilly, a leader in RNA therapeutics, is an important validation of our Axiomer RNA editing platform, and expands the application of our technology beyond our core therapeutic area focus of genetic eye disease, to potentially benefit patients with metabolic and nervous system disorders.” said Daniel A. de Boer, founder and CEO of ProQR. “Additionally, this partnership further strengthens our financial position.”

“Building from our deep scientific expertise in RNA therapies and specifically oligonucleotides, our Axiomer RNA base editing platform is uniquely positioned to target a wide range of diseases in a highly specific manner,” said Gerard Platenburg, chief innovation officer of ProQR. “Our approach uses the cell’s own editing machinery to make specific single nucleotide edits in RNA to reverse a mutation. With broad applicability, and a leading patent portfolio in the ADAR editing space, this platform represents an important strategic opportunity and has significant potential to target diseases otherwise thought untreatable.”

The companies will collaborate to develop up to five targets. Under the terms of the agreement, ProQR will receive $50 million consisting of an upfront payment of $20 million, as well as an equity investment in its ordinary shares of $30 million. ProQR is also eligible to receive up to approximately $1.25 billion for development, regulatory and commercialization milestones, as well as tiered royalties of up to mid-single digit percentage on product sales.

About Axiomer

ProQR is pioneering a next-generation RNA editing technology called Axiomer, which could potentially yield a new class of medicines for genetic diseases. Axiomer “Editing Oligonucleotides”, or EONs, induce single nucleotide changes to RNA in a highly specific and targeted way using the cell’s own molecular machinery. The Axiomer EONs are designed to recruit an endogenously expressed RNA editing system called ADAR, which can direct the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G). There are over 20,000 G to A mutations known to cause human disease that could be reversed using this novel technology.