Lilly To Try ProQR’s RNA-Editing Technology In Liver, Neurology Indications

ProQR’s Axiomer platform can enable single nucleotide RNA editing, an approach that could address thousands of disease-causing genetic mutations, the company says.

Innovation concept presented by a consultant in management on webinar screen
Lilly decides to tap into ProQR's RNA-editing technology

In its second deal around an RNA-based approach to drug development this year, Eli Lilly and Company agreed on 8 September to partner with ProQR Therapeutics N.V. on an effort to develop five novel therapies derived from RNA editing for liver and neurology indications. Lilly is paying the Leiden, the Netherlands-based firm $50m up front - $20m in cash and $30m in equity investment – with potential for up to $1.25bn in milestones to ProQR plus sales royalties.

In May, Lilly unveiled a deal with a similar financial structure – modest upfront payment and significant earnout potential – with MiNA Therapeutics Ltd. for small activating RNA (saRNA) therapeutics that would address difficult-to-drug indications by a process of restoring normal functioning of specific genes, rather than silencing them as RNA-interference therapies do

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