QR-4210
Usher Syndrome (USH2A exon 13 mutations)
Phase 2/3Development Halted
Key Facts
Indication
Usher Syndrome (USH2A exon 13 mutations)
Phase
Phase 2/3
Status
Development Halted
Company
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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