ProQR has announced a global licensing and research collaboration with Eli Lilly “focused on the discovery, development and commercialization of potential new medicines for genetic disorders in the liver and nervous system.” The companies plan to develop up to five targets using ProQR’s RNA editing platform technology.
ProQR is a Netherlands-based and NASDAQ-listed biotech which has focused to date on developing antisense therapies for inherited retinal eye diseases.
Under the terms of the deal, ProQR will initially receive $50 million from Eli Lilly, consisting of an upfront payment of $20 million and an equity investment of $30 million through ordinary shares. An additional $1.25 billion is payable if certain development, regulatory and commercialization targets are achieved, as well as tiered, single-digit royalties.
Commenting on the deal, a spokesperson for Eli Lilly said
"RNA editing is an exciting emerging technology, which allows transient, reversible editing, which in some indications may be an extremely attractive therapeutic approach."
Given the growing safety concerns associated with “once and done” AAV vector- delivered gene therapies, it is not surprising that reversible editing is now being viewed as “an extremely attractive therapeutic approach”.
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