RARE Daily

Lilly and ProQR Expand RNA Editing Collaboration

December 22, 2022

Eli Lilly and ProQR Therapeutics have expanded their licensing and collaboration agreement focused on the discovery, development, and commercialization of new genetic medicines.

Photo: Daniel de Boer, founder and CEO of ProQR

Originally announced in September 2021, the collaboration applied ProQR’s proprietary Axiomer RNA editing platform to target disorders of the liver and nervous system. Through the course of work to date, advances in the platform have significantly increased editing efficiency and refined biodistribution in both the liver and nervous system, opening up new potential applications to not only correct known mutations, but also introduce protective variants in specific transcripts.

Through this expanded collaboration, Lilly and ProQR will explore further applications of the Axiomer platform to unlock new innovative treatments for people living with diseases with high unmet medical need.

ProQR’s next-generation RNA base editing technology, Axiomer, 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.

“Discovering and developing the medicines of tomorrow takes time, sustained innovation and most importantly, collaboration,” said Andrew Adams, Lilly senior vice president of genetic medicine and co-director of the Institute for Genetic Medicine. “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.”

Under the terms of the expanded agreement, Lilly will gain access to additional targets in the central nervous system and peripheral nervous system with ProQR’s Axiomer platform. ProQR will receive $75 million consisting of an upfront payment, as well as an equity investment. Lilly will have the ability to exercise an option to further expand the partnership for a consideration of $50 million. In addition, Lilly can elect to provide ProQR with access to the company’s proprietary delivery technology for its wholly owned pipeline. With the expanded agreement, in total, ProQR is eligible to receive up to approximately $3.75 billion in research, development and commercialization milestones, as well as tiered royalties of up to mid-single digit percentage on product sales.

“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,” said Daniel de Boer, founder and CEO of ProQR.

Author: Rare Daily Staff

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