ProQR Therapeutics founder, Daniel de Boer | Image credit: ProQR
Netherlands-based ProQR Therapeutics, a biotechnology company, and US-based Eli Lilly (Lilly), announced on Thursday, December 22 that they are expanding their license and collaboration agreement for the discovery, development and commercialization of new genetic medicines.
ProQR is focused on the development of drugs to treat genetic disorders. Eli Lilly engages in the discovery, development, production and sale of products in the pharmaceutical segment.
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The aim of this collaboration
According to a statement, the partnership will assist in the discovery and development of additional assets targeting high-conviction targets using ProQR’s Axiomer technology.
Founded in 2012 by Daniel A. de Boer, ProQR Therapeutics claims to change lives through the creation of transformative RNA therapies. The company is pioneering a next-generation RNA technology called Axiomer, “which uses a cell’s own editing machinery called ADAR to make specific single-nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially provide a new class of drugs for both rare and widespread diseases with unmet needs.”
The development, first announced in September 2021, used ProQR’s patented Axiomer RNA editing tool to target liver and neurological system diseases. And according to the company, advances in the platform have increased editing efficiency and refined biodistribution in both the liver and nervous system. This has opened doors to new potential applications for not only fixing known errors but also inserting protective variants into specific transcripts.
Lilly and ProQR will explore other applications of the Axiomer platform to unlock new breakthrough medicines for individuals living with high unmet medical need diseases through this expanded relationship.
Founder and CEO of ProQR, Daniel A. de Boer, says: “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 impacting the lives of patients together with Lilly.”
ProQR to receive $75 million
The collaboration gives Lilly access to additional targets in the central and peripheral nervous system via ProQR’s Axiomer platform. ProQR will receive $75 million in total, including an upfront payment and an equity investment. For a contribution of 50 million dollars, Lilly will be able to exercise an option to extend the collaboration.
Lilly may also choose to grant ProQR access to the company’s patented delivery technology for its wholly-owned pipeline.
Under the September 2021 agreement, and today’s expanded agreement with Lilly, ProQR is eligible to receive up to nearly $3.75 billion in research, development and commercialization milestones, as well as tiered royalties of up to a single-digit percentage on the product. sale.
About Eli Lilly
Lilly claims to combine care and innovation to develop medicines that improve the lives of people globally. The company has been pioneering life-changing discoveries for nearly 150 years, and its medicines currently help more than 47 million people.
The company advances new discoveries to solve some of the most pressing health problems facing the world, including diabetes treatment; treat obesity and reduce its most harmful long-term effects; promote the fight against Alzheimer’s disease; addressing some of the most crippling immune system disorders; and develop treatments for the most aggressive forms of cancer.
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