ProQR Therapeutics N.V. (NASDAQ: PRQR) has announced a worldwide licensing and research agreement with Eli Lilly and Company to discover, produce, and commercial possible new drugs for genetic nervous and liver system disorders. As per the agreements, the companies will utilize ProQr’s novel Axiomer RNA editing platform in advancing new drug candidates towards clinical development and marketing.
Axiomer allows editing of RNA nucleotides
The Axiomer RNA editing tech allows editing of single RNA nucleotides in a highly specific and targeted way. Then the platform is based on oligonucleotides editing intended to recruit certain endogenous ADAR enzymes to select adenosine in a disease-related RNA.
Lilly VP for New Therapeutic Modalities Andrew Adams said, “RNA editing is an exciting emerging technology, which allows transient, reversible editing, which in some indications may be an extremely attractive therapeutic approach. 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.”
ProQR to receive $50 million payment
The companies will partner in developing five drug candidates. As per the agreement terms, ProQR will be eligible for a$50 million payment, including a $20 million upfront payment and a $30 million equity investment in its common shares. Also, the company will be eligible for $1.25 billion in regulatory and commercial milestones and tiered milestones of around mid-single-digit percent in sales.
PorQR Chief Innovation Officer said, “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. 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.”
