AX-1416
Undisclosed Ophthalmic Target
PreclinicalActive
Key Facts
About ProQR
ProQR Therapeutics is a mission-driven, clinical-stage biotechnology company dedicated to developing life-changing RNA therapies for severe rare and common genetic diseases. Its core achievement is the invention of the Axiomer® RNA editing platform, a next-generation technology enabling precise single-nucleotide correction, which has attracted a major multi-target collaboration with Eli Lilly. The company's strategy is to advance its internal pipeline in ophthalmology while leveraging partnerships to expand the application of Axiomer into new therapeutic areas like the liver and central nervous system, aiming to establish clinical proof-of-concept and build a sustainable, platform-based biotech.
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